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Google Keyword Rankings for : niemann pick disease cure
1
Niemann–Pick type C pathogenesis and treatment - NCBI - NIH
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3050622/
Miglustat is the only currently approved therapy for NP-C disease and clearly slows disease progression with limited side effects [35–38]. Nevertheless, it is ...
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https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3050622/
Miglustat is the only currently approved therapy for NP-C disease and clearly slows disease progression with limited side effects [35–38]. Nevertheless, it is ...
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2
Niemann-Pick disease treatment: a systematic review of ...
https://atm.amegroups.com/article/view/8611/9338
At present, there is no cure for NP disease and potential therapies should be addressed. A main goal is to develop treatments in order to minimize both general ...
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https://atm.amegroups.com/article/view/8611/9338
At present, there is no cure for NP disease and potential therapies should be addressed. A main goal is to develop treatments in order to minimize both general ...
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3
Niemann-Pick disease: Causes, symptoms, treatment, and more
https://www.medicalnewstoday.com/articles/niemann-pick-disease
Niemann-Pick disease is a rare genetic disorder that renders the body incapable of metabolizing cholesterol and other lipids inside cells.
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https://www.medicalnewstoday.com/articles/niemann-pick-disease
Niemann-Pick disease is a rare genetic disorder that renders the body incapable of metabolizing cholesterol and other lipids inside cells.
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4
Niemann Pick Disease Type C - NORD (National Organization ...
https://rarediseases.org/rare-diseases/niemann-pick-disease-type-c/
Several therapies have been studied or are currently being studied to assess their long-term safety and effectiveness as potential treatments for individuals ...
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https://rarediseases.org/rare-diseases/niemann-pick-disease-type-c/
Several therapies have been studied or are currently being studied to assess their long-term safety and effectiveness as potential treatments for individuals ...
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5
Niemann-Pick Disease | Mount Sinai - New York
https://www.mountsinai.org/care/genetics/services/lysosomal-storage-disease/disease-information/niemann-pick
There is no specific treatment or approved medical therapy for NP-B. However, clinical trials are currently in process to evaluate if a new investigational drug ...
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https://www.mountsinai.org/care/genetics/services/lysosomal-storage-disease/disease-information/niemann-pick
There is no specific treatment or approved medical therapy for NP-B. However, clinical trials are currently in process to evaluate if a new investigational drug ...
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6
First therapy to treat two types of Niemann-Pick disease, a rare ...
https://www.ema.europa.eu/en/news/first-therapy-treat-two-types-niemann-pick-disease-rare-genetic-metabolic-disorder
Xenpozyme is the first ASMD-specific treatment. This medicine is an enzyme replacement therapy, developed to replace patients' deficient or ...
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https://www.ema.europa.eu/en/news/first-therapy-treat-two-types-niemann-pick-disease-rare-genetic-metabolic-disorder
Xenpozyme is the first ASMD-specific treatment. This medicine is an enzyme replacement therapy, developed to replace patients' deficient or ...
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7
Niemann-Pick Disease Type C - Boston Children's Hospital
https://www.childrenshospital.org/conditions/niemann-pick-disease-type-c
What are the symptoms of Niemann-Pick disease type C? · enlarged liver and spleen (hepatosplenomegaly) · difficulty coordinating movement (ataxia) · abnormal eye ...
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https://www.childrenshospital.org/conditions/niemann-pick-disease-type-c
What are the symptoms of Niemann-Pick disease type C? · enlarged liver and spleen (hepatosplenomegaly) · difficulty coordinating movement (ataxia) · abnormal eye ...
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8
Niemann-Pick Disease (NP) Information - Cleveland Clinic
https://my.clevelandclinic.org/health/articles/6059-niemann-pick-disease-np
There is currently no cure for Niemann-Pick disease. Treatment is supportive. Children usually die from infection or progressive neurological ...
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https://my.clevelandclinic.org/health/articles/6059-niemann-pick-disease-np
There is currently no cure for Niemann-Pick disease. Treatment is supportive. Children usually die from infection or progressive neurological ...
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9
Niemann-Pick Disease - Child Neurology Foundation
https://www.childneurologyfoundation.org/disorder/niemann-pick-disease/
There is currently no cure for Niemann-Pick disease. Treatment is supportive. Children usually die from infection or progressive neurological loss. There is ...
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https://www.childneurologyfoundation.org/disorder/niemann-pick-disease/
There is currently no cure for Niemann-Pick disease. Treatment is supportive. Children usually die from infection or progressive neurological loss. There is ...
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10
Consensus clinical management guidelines for Niemann-Pick ...
https://ojrd.biomedcentral.com/articles/10.1186/s13023-018-0785-7
There is as yet no disease-specific curative therapy available, and the disease usually progresses to premature death. The mainstay of ...
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https://ojrd.biomedcentral.com/articles/10.1186/s13023-018-0785-7
There is as yet no disease-specific curative therapy available, and the disease usually progresses to premature death. The mainstay of ...
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11
Niemann-Pick disease: MedlinePlus Genetics
https://medlineplus.gov/genetics/condition/niemann-pick-disease/
Individuals with Niemann-Pick disease types C1 and C2 have problems with speech and swallowing that worsen over time, eventually interfering ...
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https://medlineplus.gov/genetics/condition/niemann-pick-disease/
Individuals with Niemann-Pick disease types C1 and C2 have problems with speech and swallowing that worsen over time, eventually interfering ...
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12
Niemann-Pick Disease: Causes, Symptoms & Diagnosis
https://www.healthline.com/health/niemann-pick-disease
Several treatment options, including bone marrow transplants, enzyme replacement therapy, and gene therapy have been used. Research is ongoing to determine the ...
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https://www.healthline.com/health/niemann-pick-disease
Several treatment options, including bone marrow transplants, enzyme replacement therapy, and gene therapy have been used. Research is ongoing to determine the ...
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13
Niemann-Pick type C: contemporary diagnosis and treatment ...
https://pn.bmj.com/content/19/5/420
Although there is no cure for this condition, cohort studies and randomised controlled trials have identified miglustat, a substrate reduction therapy, as a ...
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https://pn.bmj.com/content/19/5/420
Although there is no cure for this condition, cohort studies and randomised controlled trials have identified miglustat, a substrate reduction therapy, as a ...
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14
Overview of Niemann-Pick disease - UpToDate
https://www.uptodate.com/contents/overview-of-niemann-pick-disease
Niemann-Pick disease (NPD) is a group of autosomal recessive disorders associated with splenomegaly, variable neurologic deficits, ...
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https://www.uptodate.com/contents/overview-of-niemann-pick-disease
Niemann-Pick disease (NPD) is a group of autosomal recessive disorders associated with splenomegaly, variable neurologic deficits, ...
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15
Niemann-Pick disease type C2 - Myriad Genetics
https://myriad.com/womens-health/diseases/niemann-pick-disease-type-c2/
At this time, there is no cure for Niemann-Pick disease type C. Treatment focuses on managing symptoms with medication for seizures, sedatives for sleep ...
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https://myriad.com/womens-health/diseases/niemann-pick-disease-type-c2/
At this time, there is no cure for Niemann-Pick disease type C. Treatment focuses on managing symptoms with medication for seizures, sedatives for sleep ...
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16
Niemann-Pick Disease - Children's Health Issues
https://www.msdmanuals.com/home/children-s-health-issues/hereditary-metabolic-disorders/niemann-pick-disease
Niemann-Pick disease cannot be cured. There are different types of inherited disorders ...
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https://www.msdmanuals.com/home/children-s-health-issues/hereditary-metabolic-disorders/niemann-pick-disease
Niemann-Pick disease cannot be cured. There are different types of inherited disorders ...
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17
Treatment trials in Niemann-Pick type C disease | SpringerLink
https://link.springer.com/article/10.1007/s11011-021-00842-0
It is an autosomal recessive inherited disease that results from mutations in the NPC1 or NPC2 genes. The treatment efforts are focused on the ...
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https://link.springer.com/article/10.1007/s11011-021-00842-0
It is an autosomal recessive inherited disease that results from mutations in the NPC1 or NPC2 genes. The treatment efforts are focused on the ...
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18
Niemann-Pick disease type C | Alzheimer Society of Canada
https://alzheimer.ca/en/about-dementia/other-types-dementia/rare-types-dementia/niemann-pick-disease-type-c
Other symptoms · An enlarged liver or spleen, · Challenges with swallowing, · Slurred speech, · Loss of muscle strength, · Seizures, · Breathing problems, and ...
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https://alzheimer.ca/en/about-dementia/other-types-dementia/rare-types-dementia/niemann-pick-disease-type-c
Other symptoms · An enlarged liver or spleen, · Challenges with swallowing, · Slurred speech, · Loss of muscle strength, · Seizures, · Breathing problems, and ...
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19
Niemann-Pick Disease: An Approach for Diagnosis in Adulthood
https://www.cureus.com/articles/19466-niemann-pick-disease-an-approach-for-diagnosis-in-adulthood
The diagnosis is based on enzymatic tests and genetic sequencing, with the latter being the diagnostic confirmation test. No specific treatment ...
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https://www.cureus.com/articles/19466-niemann-pick-disease-an-approach-for-diagnosis-in-adulthood
The diagnosis is based on enzymatic tests and genetic sequencing, with the latter being the diagnostic confirmation test. No specific treatment ...
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20
Treating Niemann-Pick Disease Type C - YouTube
https://www.youtube.com/watch?v=dpTtP6fsgew
Rush University System for Health
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https://www.youtube.com/watch?v=dpTtP6fsgew
Rush University System for Health
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21
Niemann-Pick Disease Types, Symptoms, Treatments, Life ...
https://www.medicinenet.com/niemann_pick_disease/article.htm
There is currently no cure for Niemann-Pick disease. Treatment is supportive. Children usually die from infection or progressive neurological loss. There is ...
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https://www.medicinenet.com/niemann_pick_disease/article.htm
There is currently no cure for Niemann-Pick disease. Treatment is supportive. Children usually die from infection or progressive neurological loss. There is ...
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22
What Is Niemann-Pick Disease? - Verywell Health
https://www.verywellhealth.com/niemann-pick-disease-4158754
There are several treatments for Niemann-Pick type B, but they do not cure the disease. These include blood and platelet transfusions and ...
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https://www.verywellhealth.com/niemann-pick-disease-4158754
There are several treatments for Niemann-Pick type B, but they do not cure the disease. These include blood and platelet transfusions and ...
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23
Niemann–Pick disease - Wikipedia
https://en.wikipedia.org/wiki/Niemann%E2%80%93Pick_disease
Niemann–Pick disease is a group of severe inherited metabolic disorders, in which sphingomyelin accumulates in lysosomes in cells Niemann–Pick disease.
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https://en.wikipedia.org/wiki/Niemann%E2%80%93Pick_disease
Niemann–Pick disease is a group of severe inherited metabolic disorders, in which sphingomyelin accumulates in lysosomes in cells Niemann–Pick disease.
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24
Niemann-Pick Disease - Winchester Hospital
https://www.winchesterhospital.org/health-library/article?id=22482
Treatment. There is no cure. The goal is to manage symptoms. The methods used to do this depend on the disease type and severity. Prevention.
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https://www.winchesterhospital.org/health-library/article?id=22482
Treatment. There is no cure. The goal is to manage symptoms. The methods used to do this depend on the disease type and severity. Prevention.
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25
Niemann-Pick Disease - Newborn Screening Program - IDPH
http://www.idph.state.il.us/HealthWellness/fs/niemann_pick.htm
Individuals with Niemann-Pick disease are best treated by a team of specialists knowledgeable about the disease, who can offer supportive and symptomatic care.
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http://www.idph.state.il.us/HealthWellness/fs/niemann_pick.htm
Individuals with Niemann-Pick disease are best treated by a team of specialists knowledgeable about the disease, who can offer supportive and symptomatic care.
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26
Niemann–Pick disease - Brain Foundation
https://brainfoundation.org.au/images/stories/applicant_essays/2012_essays/Niemann-Pick_Disease-_Grubman.pdf
Type B usually begins in childhood, with milder symptoms and no brain damage. Types A and B are caused by mutations in a gene called SMPD1, which results in ...
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https://brainfoundation.org.au/images/stories/applicant_essays/2012_essays/Niemann-Pick_Disease-_Grubman.pdf
Type B usually begins in childhood, with milder symptoms and no brain damage. Types A and B are caused by mutations in a gene called SMPD1, which results in ...
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27
Recent advances in the treatment of Niemann pick disease ...
https://www.sciencedirect.com/science/article/pii/S0378517320304245
Until today there is not a cure, the current treatment is based on palliative affairs to reduce the symptoms and prevent its appearance. Among ...
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https://www.sciencedirect.com/science/article/pii/S0378517320304245
Until today there is not a cure, the current treatment is based on palliative affairs to reduce the symptoms and prevent its appearance. Among ...
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28
Niemann-Pick Disease - Newborn Screening - Baby's First Test
https://www.babysfirsttest.org/newborn-screening/conditions/niemann-pick-disease
› conditions › niemann-p...
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https://www.babysfirsttest.org/newborn-screening/conditions/niemann-pick-disease
› conditions › niemann-p...
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29
Niemann-Pick Disease | Cedars-Sinai
https://www.cedars-sinai.org/health-library/diseases-and-conditions/n/niemann-pick-disease.html
Its symptoms include variable enlargement of the liver and spleen, an increasing inability to control muscle movement, seizures, disordered muscle tone, ...
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https://www.cedars-sinai.org/health-library/diseases-and-conditions/n/niemann-pick-disease.html
Its symptoms include variable enlargement of the liver and spleen, an increasing inability to control muscle movement, seizures, disordered muscle tone, ...
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30
Niemann-Pick Disease Reference Guide - Drugs.com
https://www.drugs.com/mcd/niemann-pick
Niemann-Pick can occur at any age but mainly affects children. The disease has no known cure and is sometimes fatal. Treatment is focused on ...
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https://www.drugs.com/mcd/niemann-pick
Niemann-Pick can occur at any age but mainly affects children. The disease has no known cure and is sometimes fatal. Treatment is focused on ...
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31
Niemann-Pick Disease Type C - Rush University Medical Center
https://www.rush.edu/kids/conditions/niemann-pick-disease-type-c
Alleviating symptoms: There is currently no cure for Niemann-Pick disease type C, and there is no FDA-approved treatment to halt the disease's progression.
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https://www.rush.edu/kids/conditions/niemann-pick-disease-type-c
Alleviating symptoms: There is currently no cure for Niemann-Pick disease type C, and there is no FDA-approved treatment to halt the disease's progression.
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32
mRNA Treatment Rescues Niemann–Pick Disease Type C1 in ...
https://pubs.acs.org/doi/10.1021/acs.molpharmaceut.2c00463
Messenger RNA (mRNA) holds great potential as a disease-modifying treatment for a wide array of monogenic disorders. Niemann–Pick disease ...
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https://pubs.acs.org/doi/10.1021/acs.molpharmaceut.2c00463
Messenger RNA (mRNA) holds great potential as a disease-modifying treatment for a wide array of monogenic disorders. Niemann–Pick disease ...
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33
Niemann-Pick Disease Type C - NPUK
https://www.npuk.org/niemann-pick-disease/niemann-pick-type-c/
Niemann-Pick Type C (NPC) is a rare inherited neurodegenerative disease that affects infants, children and adults. It is caused by an accumulation of lipids ...
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https://www.npuk.org/niemann-pick-disease/niemann-pick-type-c/
Niemann-Pick Type C (NPC) is a rare inherited neurodegenerative disease that affects infants, children and adults. It is caused by an accumulation of lipids ...
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34
Niemann-Pick Disease - Focused Ultrasound Foundation
https://www.fusfoundation.org/diseases-and-conditions/niemann-pick-disease/
The primary options for treatment of Niemann-Pick Disease are primarily supportive therapies as there is no cure.
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https://www.fusfoundation.org/diseases-and-conditions/niemann-pick-disease/
The primary options for treatment of Niemann-Pick Disease are primarily supportive therapies as there is no cure.
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35
Niemann-Pick disease | Newborn Screening - HRSA
https://newbornscreening.hrsa.gov/conditions/niemann-pick-disease
causes health problems in the bone marrow, liver, spleen, and lungs. Chronic neurovisceral disease has the same symptoms as chronic visceral ...
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https://newbornscreening.hrsa.gov/conditions/niemann-pick-disease
causes health problems in the bone marrow, liver, spleen, and lungs. Chronic neurovisceral disease has the same symptoms as chronic visceral ...
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36
Niemann-Pick - Causes, Symptoms and Treatment
https://healthlibrary.askapollo.com/niemann-pick-causes-symptoms-and-treatment/
What are the symptoms of Niemann-Pick disease ? ; Swollen abdomen; Swollen lymph nodes; A red spot in the eye; Feeding difficulties and reduced ...
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https://healthlibrary.askapollo.com/niemann-pick-causes-symptoms-and-treatment/
What are the symptoms of Niemann-Pick disease ? ; Swollen abdomen; Swollen lymph nodes; A red spot in the eye; Feeding difficulties and reduced ...
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37
Correlation of age of onset and clinical severity in Niemann ...
https://www.nature.com/articles/s41598-022-06112-y
Currently, there are very limited treatment options available for NPC disease, and most potential therapies are undergoing clinical trials. Of ...
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https://www.nature.com/articles/s41598-022-06112-y
Currently, there are very limited treatment options available for NPC disease, and most potential therapies are undergoing clinical trials. Of ...
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38
Sphingomyelinase Deficiency - Medscape Reference
https://emedicine.medscape.com/article/951564-overview
Niemann-Pick disease (NPD) is a lipid storage disorder that results from the ... Signs and symptoms of sphingomyelinase deficiency.
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https://emedicine.medscape.com/article/951564-overview
Niemann-Pick disease (NPD) is a lipid storage disorder that results from the ... Signs and symptoms of sphingomyelinase deficiency.
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39
NP-C Diesease - Signs + Symptoms - Niemann-Pick Type C ...
http://www.parseghian.org/aboutnpc_diagnosis.html
While there not yet a specific treatment for NP-C, supportive therapies are available. These include medications to control seizures, abnormal posturing of ...
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http://www.parseghian.org/aboutnpc_diagnosis.html
While there not yet a specific treatment for NP-C, supportive therapies are available. These include medications to control seizures, abnormal posturing of ...
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40
UCSF Niemann-Pick Disease Clinical Trials for 2022
https://clinicaltrials.ucsf.edu/niemann-pick-disease
Do you qualify for these Niemann-Pick Disease studies? We're researching new and effective treatments.
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https://clinicaltrials.ucsf.edu/niemann-pick-disease
Do you qualify for these Niemann-Pick Disease studies? We're researching new and effective treatments.
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41
Niemann-Pick disease type C | Alzheimer's Society
https://www.alzheimers.org.uk/about-dementia/types-dementia/Niemann-Pick-disease-type-C
People who first show symptoms in late adolescence or early adulthood are more likely to experience dementia as part of the disease. The dementia symptoms ...
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https://www.alzheimers.org.uk/about-dementia/types-dementia/Niemann-Pick-disease-type-C
People who first show symptoms in late adolescence or early adulthood are more likely to experience dementia as part of the disease. The dementia symptoms ...
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42
Gene therapy shows promise for treating Niemann-Pick ...
https://www.genome.gov/news/news-release/Gene-therapy-shows-promise-for-treating-Niemann-Pick-disease-type-C1
Niemann-Pick disease is a rare and fatal disorder of the central nervous system (the brain and spinal cord) that has no cure.
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https://www.genome.gov/news/news-release/Gene-therapy-shows-promise-for-treating-Niemann-Pick-disease-type-C1
Niemann-Pick disease is a rare and fatal disorder of the central nervous system (the brain and spinal cord) that has no cure.
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43
About NPD - International Niemann-Pick Disease Alliance ...
https://www.inpda.org/about-niemann-pick-diseases/
Can Niemann-Pick diseases be treated? · ASMD Niemann Pick Type A: Unfortunately there is no cure or disease modifying treatment available for this condition.
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https://www.inpda.org/about-niemann-pick-diseases/
Can Niemann-Pick diseases be treated? · ASMD Niemann Pick Type A: Unfortunately there is no cure or disease modifying treatment available for this condition.
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44
Niemann-Pick Disease Type C | Children's Hospital Pittsburgh
https://www.chp.edu/our-services/rare-disease-therapy/conditions-we-treat/niemann-pick-disease-type-c
Children with NPC may have difficulty moving their eyes up and down and may also have jaundice, seizures, difficulty breathing, and other symptoms. Maria Luisa ...
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https://www.chp.edu/our-services/rare-disease-therapy/conditions-we-treat/niemann-pick-disease-type-c
Children with NPC may have difficulty moving their eyes up and down and may also have jaundice, seizures, difficulty breathing, and other symptoms. Maria Luisa ...
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45
Niemann Pick disease type C - Orphanet
https://www.orpha.net/consor/cgi-bin/OC_Exp.php?Expert=646
Treatment with an iminosugar inhibitor of glucosylceramide synthase (miglustat) was approved for the management of neurologic manifestations of NPDC in several ...
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https://www.orpha.net/consor/cgi-bin/OC_Exp.php?Expert=646
Treatment with an iminosugar inhibitor of glucosylceramide synthase (miglustat) was approved for the management of neurologic manifestations of NPDC in several ...
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46
Niemann-Pick disease | Handouts - MedLink Neurology
https://www.medlink.com/handouts/niemann-pick-disease
Treatment options for NPB. Research into therapies for NPB has progressed rapidly since the early 1990's. Mount Sinai School of Medicine is conducting research ...
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https://www.medlink.com/handouts/niemann-pick-disease
Treatment options for NPB. Research into therapies for NPB has progressed rapidly since the early 1990's. Mount Sinai School of Medicine is conducting research ...
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47
Entrance of novel therapies will drive Niemann-Pick type C ...
https://www.pharmaceutical-technology.com/comment/novel-therapies-niemann-pick/
The current treatment for NPC involves the use of miglustat, a glucosylceramide synthase (GCS) inhibitor, delaying the onset of neurological ...
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https://www.pharmaceutical-technology.com/comment/novel-therapies-niemann-pick/
The current treatment for NPC involves the use of miglustat, a glucosylceramide synthase (GCS) inhibitor, delaying the onset of neurological ...
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48
A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick ...
https://clinicaltrials.gov/ct2/show/NCT05163288
Condition or disease, Intervention/treatment, Phase. Niemann-Pick Disease, Type C, Drug: N-Acetyl-L-Leucine Other: Placebo, Phase 3 ...
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https://clinicaltrials.gov/ct2/show/NCT05163288
Condition or disease, Intervention/treatment, Phase. Niemann-Pick Disease, Type C, Drug: N-Acetyl-L-Leucine Other: Placebo, Phase 3 ...
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49
Arimoclomol Appears to Slow Progression of Niemann-Pick ...
https://journals.lww.com/neurotodayonline/blog/neurologytodayconferencereporterscnsannualmeeting/pages/post.aspx?PostID=48
Current treatment of Niemann-Pick Type C disease often included miglustat, which inhibits glucosylceramide synthase, and reduces the ...
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https://journals.lww.com/neurotodayonline/blog/neurologytodayconferencereporterscnsannualmeeting/pages/post.aspx?PostID=48
Current treatment of Niemann-Pick Type C disease often included miglustat, which inhibits glucosylceramide synthase, and reduces the ...
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50
Niemann-Pick Disease Article - StatPearls
https://www.statpearls.com/ArticleLibrary/viewarticle/25882
Niemann-Pick disease has negative repercussions for many organs, and with or without treatment, it has high morbidity and mortality. The ...
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https://www.statpearls.com/ArticleLibrary/viewarticle/25882
Niemann-Pick disease has negative repercussions for many organs, and with or without treatment, it has high morbidity and mortality. The ...
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51
SSA - POMS: DI 23022.475 - Niemann-Pick Disease - Type C
https://secure.ssa.gov/poms.Nsf/lnx/0423022475
There is currently no effective cure for this disease. Medicines are available to control or relieve many symptoms such as cataplexy and ...
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https://secure.ssa.gov/poms.Nsf/lnx/0423022475
There is currently no effective cure for this disease. Medicines are available to control or relieve many symptoms such as cataplexy and ...
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52
Niemann-Pick disease | medical disorder | Britannica
https://www.britannica.com/science/Niemann-Pick-disease
Symptoms consist of extreme liver and spleen enlargement, mental retardation, and a brownish-yellow skin discoloration; foamy cells containing phospholipids ...
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https://www.britannica.com/science/Niemann-Pick-disease
Symptoms consist of extreme liver and spleen enlargement, mental retardation, and a brownish-yellow skin discoloration; foamy cells containing phospholipids ...
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53
Niemann-Pick Disease Symptoms, Doctors, Treatments ...
https://www.medifind.com/conditions/niemann-pick-disease/3803
Find everything you need to know about Niemann-Pick Disease including doctors, latest advances, and ongoing clinical trials.
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https://www.medifind.com/conditions/niemann-pick-disease/3803
Find everything you need to know about Niemann-Pick Disease including doctors, latest advances, and ongoing clinical trials.
→ Check Latest Keyword Rankings ←
54
Niemann-Pick Disease - United Brain Association
https://unitedbrainassociation.org/brain-resources/niemann-pick-disease/
How Is Niemann-Pick Disease Treated? · Feeding assistance, including a feeding tube · Anti-seizure medications · Medications to improve poor muscle ...
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https://unitedbrainassociation.org/brain-resources/niemann-pick-disease/
How Is Niemann-Pick Disease Treated? · Feeding assistance, including a feeding tube · Anti-seizure medications · Medications to improve poor muscle ...
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55
Niemann Pick Disease - Causes, Type ... - Health Jade
https://healthjade.com/niemann-pick/
Niemann-Pick disease is a group of rare inherited lysosomal storage disorders that affects many body systems and can affect both children and adults. Niemann- ...
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https://healthjade.com/niemann-pick/
Niemann-Pick disease is a group of rare inherited lysosomal storage disorders that affects many body systems and can affect both children and adults. Niemann- ...
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56
Niemann-Pick - eurordis.org
https://www.eurordis.org/stories/niemann-pick/
Although there is still no cure for NP, research into this “lysosomal storage disorder” is progressing, thanks largely to fundraising efforts by support groups ...
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https://www.eurordis.org/stories/niemann-pick/
Although there is still no cure for NP, research into this “lysosomal storage disorder” is progressing, thanks largely to fundraising efforts by support groups ...
→ Check Latest Keyword Rankings ←
57
Pick's Disease - Symptoms and Causes - Penn Medicine
https://www.pennmedicine.org/for-patients-and-visitors/patient-information/conditions-treated-a-to-z/picks-disease
There is no specific treatment for FTD. Medicines may help manage mood swings. Sometimes, people with FTD take the same medicines used to treat other types of ...
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https://www.pennmedicine.org/for-patients-and-visitors/patient-information/conditions-treated-a-to-z/picks-disease
There is no specific treatment for FTD. Medicines may help manage mood swings. Sometimes, people with FTD take the same medicines used to treat other types of ...
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58
Why are people fighting over a promising treatment for a fatal ...
https://www.science.org/content/article/why-are-people-fighting-over-promising-treatment-fatal-childhood-disease
The furor surrounds the use of cyclodextrins, a family of doughnut-shaped sugar molecules, to treat Niemann-Pick type C-1 (NPC), a lethal, inherited disease ...
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https://www.science.org/content/article/why-are-people-fighting-over-promising-treatment-fatal-childhood-disease
The furor surrounds the use of cyclodextrins, a family of doughnut-shaped sugar molecules, to treat Niemann-Pick type C-1 (NPC), a lethal, inherited disease ...
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59
Marta, with Niemann Pick C, fights for a cure with gene therapy
https://fundacioncolumbus.org/marta-with-niemann-pick-c-fights-for-a-cure-with-gene-therapy/?lang=en
Currently there are no cures for this disease. There only exits a medication called Miglustat, that currently has been shown to slow down the advancement of the ...
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https://fundacioncolumbus.org/marta-with-niemann-pick-c-fights-for-a-cure-with-gene-therapy/?lang=en
Currently there are no cures for this disease. There only exits a medication called Miglustat, that currently has been shown to slow down the advancement of the ...
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60
Testing of Novel Treatment Options for Niemann-Pick Disease
https://www.centogene.com/resources/scientific-publications/testing-of-novel-treatment-options-for-niemann-pick-disease
... researchers recently investigated the potential effects of a novel candidate drug for improving neurological symptoms of Niemann-Pick disease.
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https://www.centogene.com/resources/scientific-publications/testing-of-novel-treatment-options-for-niemann-pick-disease
... researchers recently investigated the potential effects of a novel candidate drug for improving neurological symptoms of Niemann-Pick disease.
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61
Niemann-Pick | Beacon Health System
https://www.beaconhealthsystem.org/library/diseases-and-conditions/niemann-pick?content_id=CON-20257402
No cure exists for Niemann-Pick disease. No effective treatment is available to people with type A or B. For people with mild to moderate type C ...
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https://www.beaconhealthsystem.org/library/diseases-and-conditions/niemann-pick?content_id=CON-20257402
No cure exists for Niemann-Pick disease. No effective treatment is available to people with type A or B. For people with mild to moderate type C ...
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62
Niemann-Pick Disease, SMPD1-associated - JScreen
https://jscreen.org/learn-more/diseases/niemann-pick-disease/
Unfortunately there are no effective treatments for Niemann-Pick disease type A. Medical professionals can attempt to treat the symptoms through physical ...
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https://jscreen.org/learn-more/diseases/niemann-pick-disease/
Unfortunately there are no effective treatments for Niemann-Pick disease type A. Medical professionals can attempt to treat the symptoms through physical ...
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63
Potential Disease-Modifying Effects of Lithium Carbonate in ...
https://www.frontiersin.org/articles/10.3389/fphar.2021.667361/full
Background: Niemann-Pick disease type C1 (NP-C1) is a rare, ... The mean NNSS was improved after lithium treatment at 12 months (p = 0.005).
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https://www.frontiersin.org/articles/10.3389/fphar.2021.667361/full
Background: Niemann-Pick disease type C1 (NP-C1) is a rare, ... The mean NNSS was improved after lithium treatment at 12 months (p = 0.005).
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64
Niemann-Pick disease, type C - Labcorp Women's Health
https://womenshealth.labcorp.com/sites/default/files/2021-10/Niemann-Pick%20disease%20type%20C_0317.pdf
Niemann-Pick disease, type C, is an inherited disease characterized by an inability of the body to properly move cholesterol and lipids within cells. Symptoms ...
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https://womenshealth.labcorp.com/sites/default/files/2021-10/Niemann-Pick%20disease%20type%20C_0317.pdf
Niemann-Pick disease, type C, is an inherited disease characterized by an inability of the body to properly move cholesterol and lipids within cells. Symptoms ...
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65
Gene therapy shows promise for treating Niemann ... - MDLinx
https://www.mdlinx.com/article/gene-therapy-shows-promise-for-treating-niemann-pick-disease-type-c1/lfc-656
Researchers have found that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1 (NPC1), ...
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https://www.mdlinx.com/article/gene-therapy-shows-promise-for-treating-niemann-pick-disease-type-c1/lfc-656
Researchers have found that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1 (NPC1), ...
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66
Niemann-Pick Disease. Abnormal lipid Metabolism. - Patient.info
https://patient.info/doctor/niemann-pick-disease-pro
Management · There is no specific treatment for NPA or NPB. · Miglustat has been shown to decelerate neurological regression for people with NPC.
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https://patient.info/doctor/niemann-pick-disease-pro
Management · There is no specific treatment for NPA or NPB. · Miglustat has been shown to decelerate neurological regression for people with NPC.
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67
Niemann-Pick disease symptoms, treatments & forums
https://www.patientslikeme.com/conditions/niemann-pick
Niemann-Pick disease is an inherited condition involving lipid metabolism, which is the breakdown, transport, and use of fats and cholesterol in the body.
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https://www.patientslikeme.com/conditions/niemann-pick
Niemann-Pick disease is an inherited condition involving lipid metabolism, which is the breakdown, transport, and use of fats and cholesterol in the body.
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68
Niemann-Pick Disease Information - StoryMD
https://storymd.com/journal/vj636g6izj/page/7qdde1xqkq
There is currently no cure for Niemann-Pick disease. Treatment is supportive. Children usually die from infection or progressive neurological loss. There is ...
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https://storymd.com/journal/vj636g6izj/page/7qdde1xqkq
There is currently no cure for Niemann-Pick disease. Treatment is supportive. Children usually die from infection or progressive neurological loss. There is ...
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69
Niemann-Pick Disease | Concise Medical Knowledge - Lecturio
https://www.lecturio.com/concepts/niemann-pick-disease/
Severe; Presents within the 1st few months of life; Rapidly progressive neurodegenerative course; Death by 3 years of age. Signs and symptoms:.
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https://www.lecturio.com/concepts/niemann-pick-disease/
Severe; Presents within the 1st few months of life; Rapidly progressive neurodegenerative course; Death by 3 years of age. Signs and symptoms:.
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70
Diseases – NNPDF
https://nnpdf.org/diseases/
The National Niemann-Pick disease Foundation (NNPDF) supports and promotes research to find treatments and a cure for all types of Niemann-Pick disease, ...
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https://nnpdf.org/diseases/
The National Niemann-Pick disease Foundation (NNPDF) supports and promotes research to find treatments and a cure for all types of Niemann-Pick disease, ...
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71
Recent Advances in the Diagnosis and Treatment of Niemann ...
https://www.researchgate.net/publication/273485495_Recent_Advances_in_the_Diagnosis_and_Treatment_of_Niemann-Pick_Disease_Type_C_in_Children_A_Guide_to_Early_Diagnosis_for_the_General_Pediatrician
› publication › 273485495_...
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https://www.researchgate.net/publication/273485495_Recent_Advances_in_the_Diagnosis_and_Treatment_of_Niemann-Pick_Disease_Type_C_in_Children_A_Guide_to_Early_Diagnosis_for_the_General_Pediatrician
› publication › 273485495_...
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72
Niemann Pick Disease Market Size Share Growth ...
https://www.datamintelligence.com/research-report/niemann-pick-disease-market
The disease has no known cure and can be fatal in some cases. The goal of treatment is to help people cope with their symptoms. Researchers at ...
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https://www.datamintelligence.com/research-report/niemann-pick-disease-market
The disease has no known cure and can be fatal in some cases. The goal of treatment is to help people cope with their symptoms. Researchers at ...
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73
Gene Therapy for Niemann-Pick Type a Diseases Moves ...
https://www.technologynetworks.com/biopharma/news/gene-therapy-for-niemann-pick-type-a-diseases-moves-forward-323100
Niemann-Pick disease primarily affects children, has no known cure and is often fatal. It's caused by mutations in a gene that causes a ...
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https://www.technologynetworks.com/biopharma/news/gene-therapy-for-niemann-pick-type-a-diseases-moves-forward-323100
Niemann-Pick disease primarily affects children, has no known cure and is often fatal. It's caused by mutations in a gene that causes a ...
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74
Gender-Specific Effects of Two Treatment Strategies in ... - MDPI
https://www.mdpi.com/1422-0067/22/5/2539
In a mouse model of Niemann-Pick disease type C1 (NPC1), a combination therapy (COMBI) of miglustat (MIGLU), the neurosteroid allopregnanolone (ALLO) and ...
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https://www.mdpi.com/1422-0067/22/5/2539
In a mouse model of Niemann-Pick disease type C1 (NPC1), a combination therapy (COMBI) of miglustat (MIGLU), the neurosteroid allopregnanolone (ALLO) and ...
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75
TEACHING CHILDREN WITH NIEMANN-PICK DISEASE - ERIC
https://files.eric.ed.gov/fulltext/EJ1061921.pdf
described by Niemann and Pick (National Organization for Rare Disorders. [NORD], 2006). Regardless of the subtype, symptoms include “yellow dis-.
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https://files.eric.ed.gov/fulltext/EJ1061921.pdf
described by Niemann and Pick (National Organization for Rare Disorders. [NORD], 2006). Regardless of the subtype, symptoms include “yellow dis-.
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76
The Rare Disease Report Podcast: Niemann-Pick Disease
https://www.hcplive.com/view/rare-disease-report-podcast-niemann-pick-disease
There is no cure for this disease. Quotes of Interest. Historically, Niemann-Pick was discovered as a disease back in 1900 when a pediatrician ...
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https://www.hcplive.com/view/rare-disease-report-podcast-niemann-pick-disease
There is no cure for this disease. Quotes of Interest. Historically, Niemann-Pick was discovered as a disease back in 1900 when a pediatrician ...
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77
Understanding and Treating Niemann–Pick Type C Disease
https://pdfs.semanticscholar.org/286e/ad8a179fcb971197bf0fe85c1b23d3df4d39.pdf
Niemann–Pick type C (NPC) is a prime example for a disease that lacks a curative therapy despite substantial breakthroughs. This rare, fatal, ...
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https://pdfs.semanticscholar.org/286e/ad8a179fcb971197bf0fe85c1b23d3df4d39.pdf
Niemann–Pick type C (NPC) is a prime example for a disease that lacks a curative therapy despite substantial breakthroughs. This rare, fatal, ...
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78
Niemann Pick Disease - Causes, Type ... - Health Jade
https://healthjade.net/niemann-pick/
There is currently no specific approved treatment, or cure, for Niemann-Pick Disease Type B. However, patients will benefit from palliative treatments ( ...
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https://healthjade.net/niemann-pick/
There is currently no specific approved treatment, or cure, for Niemann-Pick Disease Type B. However, patients will benefit from palliative treatments ( ...
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79
Animal models for Niemann-Pick type C: implications for drug ...
https://www.tandfonline.com/doi/full/10.1080/17460441.2019.1588882
Introduction: Niemann-Pick type C (NPC) is a neurovisceral, progressively detrimental lysosomal storage disease with very limited therapeutic options and no ...
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https://www.tandfonline.com/doi/full/10.1080/17460441.2019.1588882
Introduction: Niemann-Pick type C (NPC) is a neurovisceral, progressively detrimental lysosomal storage disease with very limited therapeutic options and no ...
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80
Pick's Disease - Common Symptoms and Causes - WebMD
https://www.webmd.com/alzheimers/guide/picks-disease
There's no cure for Pick's disease, and medications can't slow it down. It can progress slowly, but usually it steadily gets worse over time.
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https://www.webmd.com/alzheimers/guide/picks-disease
There's no cure for Pick's disease, and medications can't slow it down. It can progress slowly, but usually it steadily gets worse over time.
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81
Efficacy of N-acetylcysteine in phenotypic suppression of ...
https://academic.oup.com/hmg/article/22/17/3508/572848
Niemann–Pick disease, type C1 (NPC1), which arises from a mutation in the NPC1 ... The Npc1−/− neurological symptoms initially present at 6–7 weeks of age ...
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https://academic.oup.com/hmg/article/22/17/3508/572848
Niemann–Pick disease, type C1 (NPC1), which arises from a mutation in the NPC1 ... The Npc1−/− neurological symptoms initially present at 6–7 weeks of age ...
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82
Valley boy battling rare disease getting stronger
https://www.abc15.com/news/state/valley-baby-showing-great-progress-with-experimental-niemann-pick-type-c-disease-drug-treatments
Woodrow Mille suffers from Niemann-Pick Type C disease, ... hard to get Woodrow enrolled into an experimental drug treatment program, ...
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https://www.abc15.com/news/state/valley-baby-showing-great-progress-with-experimental-niemann-pick-type-c-disease-drug-treatments
Woodrow Mille suffers from Niemann-Pick Type C disease, ... hard to get Woodrow enrolled into an experimental drug treatment program, ...
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83
EMA Recommends Approval of Niemann-Pick Disease Types ...
https://www.pharmtech.com/view/ema-recommends-approval-of-niemann-pick-disease-types-a-and-b-therapy
Currently, there are no approved medicines that modify or slow the progression of the disease within the EU, meaning that patients are only able ...
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https://www.pharmtech.com/view/ema-recommends-approval-of-niemann-pick-disease-types-a-and-b-therapy
Currently, there are no approved medicines that modify or slow the progression of the disease within the EU, meaning that patients are only able ...
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84
Niemann-Pick disease type C: testing a potential new drug ...
https://action.org.uk/research/niemann-pick-disease-type-c-testing-potential-new-drug-treatment
Primarily affecting children, NPC is a progressive disease and sadly many do not survive into adulthood. Although the age of diagnosis and how ...
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https://action.org.uk/research/niemann-pick-disease-type-c-testing-potential-new-drug-treatment
Primarily affecting children, NPC is a progressive disease and sadly many do not survive into adulthood. Although the age of diagnosis and how ...
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85
What Is Acid Sphingomyelinase Deficiency or Niemann-Pick ...
https://themighty.com/topic/niemann-pick-disease/what-is-acid-sphingomyelinase-deficiency/
Physical therapy is also an important part of treatment to help maintain mobility as long as possible. People with Niemann-Pick disease need to ...
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https://themighty.com/topic/niemann-pick-disease/what-is-acid-sphingomyelinase-deficiency/
Physical therapy is also an important part of treatment to help maintain mobility as long as possible. People with Niemann-Pick disease need to ...
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86
Niemann–Pick Disease: Symptoms, Types and Treatment of ...
https://gomedii.com/blogs/english/health-a2z/niemann-pick-disease-symptoms-types-and-treatment/
› blogs › english › niemann-pick-d...
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https://gomedii.com/blogs/english/health-a2z/niemann-pick-disease-symptoms-types-and-treatment/
› blogs › english › niemann-pick-d...
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87
Niemann–Pick Disease Type C | CheckRare
https://checkrare.com/niemann-pick-disease-type-c/
Niemann-Pick disease type C (NPC) is a disabling, lysosomal-storage disorder that has been diagnosed prenatally, neonatally, ...
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https://checkrare.com/niemann-pick-disease-type-c/
Niemann-Pick disease type C (NPC) is a disabling, lysosomal-storage disorder that has been diagnosed prenatally, neonatally, ...
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88
VTS-270-303 Niemann-Pick Disease, Type C
https://cure4thekids.org/vts-270-303-niemann-pick-disease-type-c/
Genetics. Protocol:VTS-270-303. Condition or Disease: Niemann-Pick Disease, Type C. Study Type: Interventional (Clinical Trial). Status: Opening Mid 2019.
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https://cure4thekids.org/vts-270-303-niemann-pick-disease-type-c/
Genetics. Protocol:VTS-270-303. Condition or Disease: Niemann-Pick Disease, Type C. Study Type: Interventional (Clinical Trial). Status: Opening Mid 2019.
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89
Niemann-Pick Type C Disease—Pathophysiology and Future ...
https://touchneurology.com/alzheimers-disease-dementia/journal-articles/niemann-pick-type-c-disease-pathophysiology-and-future-perspectives-for-treatment/
Niemann-Pick Type C Disease—Pathophysiology and Future Perspectives for Treatment. Published Online: June 4th 2011 US Neurology, ...
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https://touchneurology.com/alzheimers-disease-dementia/journal-articles/niemann-pick-type-c-disease-pathophysiology-and-future-perspectives-for-treatment/
Niemann-Pick Type C Disease—Pathophysiology and Future Perspectives for Treatment. Published Online: June 4th 2011 US Neurology, ...
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90
Study offers new insights into "Niemann-Pick type C disease"
https://www.news-medical.net/news/20210224/Study-offers-new-insights-into-Niemann-Pick-type-C-disease.aspx
NPC mainly manifests in childhood. Current therapies can alleviate symptoms somewhat, but cannot sustainably halt progression of the disease.
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https://www.news-medical.net/news/20210224/Study-offers-new-insights-into-Niemann-Pick-type-C-disease.aspx
NPC mainly manifests in childhood. Current therapies can alleviate symptoms somewhat, but cannot sustainably halt progression of the disease.
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91
Advancing diagnosis and treatment of Niemann-Pick C ...
https://www.explorationpub.com/Journals/ent/Article/100412
Niemann-Pick C disease is a rare neurodegenerative, lysosomal storage disease caused by accumulation of unesterified cholesterol. Diagnosis of the disease ...
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https://www.explorationpub.com/Journals/ent/Article/100412
Niemann-Pick C disease is a rare neurodegenerative, lysosomal storage disease caused by accumulation of unesterified cholesterol. Diagnosis of the disease ...
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92
Niemann–Pick Disease Type B – A Case Report
http://jmscr.igmpublication.org/home/index.php/current-issue/2058-niemann-pick-disease-type-b-a-case-report
There is no specific treatment for Niemann-Pick Disease. Type A is a severe form of disease, with average life span of 18 months.
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http://jmscr.igmpublication.org/home/index.php/current-issue/2058-niemann-pick-disease-type-b-a-case-report
There is no specific treatment for Niemann-Pick Disease. Type A is a severe form of disease, with average life span of 18 months.
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93
Long‐term survival outcomes of patients with Niemann‐Pick ...
https://onlinelibrary.wiley.com/doi/abs/10.1002/jimd.12245
Miglustat has been indicated for the treatment of Niemann-Pick disease type C (NP-C) since 2009. The aim of this observational study was to ...
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https://onlinelibrary.wiley.com/doi/abs/10.1002/jimd.12245
Miglustat has been indicated for the treatment of Niemann-Pick disease type C (NP-C) since 2009. The aim of this observational study was to ...
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94
Searching for a cure for Niemann-Pick Type C using C. elegans
https://invivobiosystems.com/disease-modeling/searching-cure-niemann-pick-type-c-using-c-elegans/
Because it is an orphan disease, NPC requires a low-cost approach for drug development. There is currently no cure for NPC. We recently began a research ...
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https://invivobiosystems.com/disease-modeling/searching-cure-niemann-pick-type-c-using-c-elegans/
Because it is an orphan disease, NPC requires a low-cost approach for drug development. There is currently no cure for NPC. We recently began a research ...
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95
Bike Ride for a Cure raises funds for Niemann-Pick Type-C ...
https://science.nd.edu/news/bike-ride-for-a-cure-raises-funds-for-niemann-pick-type-c-research-and-support/
Niemann-Pick is a rare, inherited disease that affects the body's ability to metabolize cholesterol and other lipids within cells. These cells ...
→ Check Latest Keyword Rankings ←
https://science.nd.edu/news/bike-ride-for-a-cure-raises-funds-for-niemann-pick-type-c-research-and-support/
Niemann-Pick is a rare, inherited disease that affects the body's ability to metabolize cholesterol and other lipids within cells. These cells ...
→ Check Latest Keyword Rankings ←
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