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Google Keyword Rankings for : dmd treatment 2013
1
Eteplirsen for the treatment of Duchenne muscular dystrophy
https://pubmed.ncbi.nlm.nih.gov/23907995/
Eteplirsen restored dystrophin in the 30 and 50 mg/kg/wk cohorts, and in subsequently treated, placebo-controlled subjects. Duration, more than dose, ...
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https://pubmed.ncbi.nlm.nih.gov/23907995/
Eteplirsen restored dystrophin in the 30 and 50 mg/kg/wk cohorts, and in subsequently treated, placebo-controlled subjects. Duration, more than dose, ...
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2
Eteplirsen for the treatment of Duchenne muscular dystrophy
https://onlinelibrary.wiley.com/doi/abs/10.1002/ana.23982
DMD boys aged 7 to 13 years, with confirmed deletions correctable by skipping exon 51 and ability to walk 200 to 400 m on 6 MWT, were randomized ...
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https://onlinelibrary.wiley.com/doi/abs/10.1002/ana.23982
DMD boys aged 7 to 13 years, with confirmed deletions correctable by skipping exon 51 and ability to walk 200 to 400 m on 6 MWT, were randomized ...
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3
Medical Management - Duchenne Muscular Dystrophy (DMD)
https://www.mda.org/disease/duchenne-muscular-dystrophy/medical-management
The corticosteroids prednisone and deflazacort are beneficial in the treatment of DMD. The FDA on Feb. 9, 2017, approved deflazacort (brand name Emflaza), an ...
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https://www.mda.org/disease/duchenne-muscular-dystrophy/medical-management
The corticosteroids prednisone and deflazacort are beneficial in the treatment of DMD. The FDA on Feb. 9, 2017, approved deflazacort (brand name Emflaza), an ...
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4
Duchenne and Becker muscular dystrophy - UpToDate
https://www.uptodate.com/contents/duchenne-and-becker-muscular-dystrophy-management-and-prognosis
Glucocorticoids are the mainstay of pharmacologic treatment for DMD because of their beneficial effects for improving motor function and ...
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https://www.uptodate.com/contents/duchenne-and-becker-muscular-dystrophy-management-and-prognosis
Glucocorticoids are the mainstay of pharmacologic treatment for DMD because of their beneficial effects for improving motor function and ...
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5
The burden, epidemiology, costs and treatment for Duchenne ...
https://ojrd.biomedcentral.com/articles/10.1186/s13023-017-0631-3
There is no cure for DMD and current treatment options focus on ... For Schreiber-Katz [23], 2013 total direct medical costs ranged from ...
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https://ojrd.biomedcentral.com/articles/10.1186/s13023-017-0631-3
There is no cure for DMD and current treatment options focus on ... For Schreiber-Katz [23], 2013 total direct medical costs ranged from ...
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6
Eteplirsen in the treatment of Duchenne muscular dystrophy
https://www.dovepress.com/eteplirsen-in-the-treatment-of-duchenne-muscular-dystrophy-peer-reviewed-fulltext-article-DDDT
Figure 1 Eteplirsen is an exon-skipping therapeutic. Notes: Eteplirsen (green bar) specifically recognizes exon 51 of the DMD gene. Upon binding ...
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https://www.dovepress.com/eteplirsen-in-the-treatment-of-duchenne-muscular-dystrophy-peer-reviewed-fulltext-article-DDDT
Figure 1 Eteplirsen is an exon-skipping therapeutic. Notes: Eteplirsen (green bar) specifically recognizes exon 51 of the DMD gene. Upon binding ...
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7
Finding the Optimum Regimen for Duchenne Muscular ...
https://www.clinicaltrials.gov/ct2/show/NCT01603407
The FOR DMD study will enroll boys with DMD ages 4-7. ... Study Start Date : January 2013 ... Deflazacort treatment of Duchenne muscular dystrophy.
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https://www.clinicaltrials.gov/ct2/show/NCT01603407
The FOR DMD study will enroll boys with DMD ages 4-7. ... Study Start Date : January 2013 ... Deflazacort treatment of Duchenne muscular dystrophy.
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8
guideline-clinical-investigation-medicinal-products-treatment ...
https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-clinical-investigation-medicinal-products-treatment-duchenne-becker-muscular-dystrophy_en.pdf
21 February 2013. Start of public consultation. 1st March 2013 ... the diagnostic process of DMD/BMD in treatment centres in the EU.
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https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-clinical-investigation-medicinal-products-treatment-duchenne-becker-muscular-dystrophy_en.pdf
21 February 2013. Start of public consultation. 1st March 2013 ... the diagnostic process of DMD/BMD in treatment centres in the EU.
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9
Duchenne muscular dystrophy | Nature Reviews Disease ...
https://www.nature.com/articles/s41572-021-00248-3
In general, neonatal screening is considered for neonatal-onset disorders for which early treatment shows strong evidence of improved outcome.
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https://www.nature.com/articles/s41572-021-00248-3
In general, neonatal screening is considered for neonatal-onset disorders for which early treatment shows strong evidence of improved outcome.
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10
The Duchenne Muscular Dystrophy (DMD) Continuum
https://www.neurologylive.com/view/the-duchenne-muscular-dystrophy-dmd-continuum-the-case-for-early-intervention-and-long-term-treatment
As such, international care guidelines for DMD recommend a coordinated, multidisciplinary approach to care, and continuous treatment with ...
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https://www.neurologylive.com/view/the-duchenne-muscular-dystrophy-dmd-continuum-the-case-for-early-intervention-and-long-term-treatment
As such, international care guidelines for DMD recommend a coordinated, multidisciplinary approach to care, and continuous treatment with ...
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11
Safety, Tolerability, and Efficacy of Viltolarsen in Boys With ...
https://jamanetwork.com/journals/jamaneurology/fullarticle/2766519
Findings Results of this 4-week randomized clinical trial for safety followed by a 20-week open-label treatment period in 16 patients with DMD ...
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https://jamanetwork.com/journals/jamaneurology/fullarticle/2766519
Findings Results of this 4-week randomized clinical trial for safety followed by a 20-week open-label treatment period in 16 patients with DMD ...
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12
Prognostic indicators of disease progression in Duchenne ...
https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0265879
All-cause mortality and cardiovascular outcomes with prophylactic steroid therapy in Duchenne muscular dystrophy. J Am Coll Cardiol. 2013 ...
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https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0265879
All-cause mortality and cardiovascular outcomes with prophylactic steroid therapy in Duchenne muscular dystrophy. J Am Coll Cardiol. 2013 ...
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13
Early Diagnosis and Treatment – The Use of Ataluren in the ...
https://touchneurology.com/neuromuscular-diseases/journal-articles/early-diagnosis-and-treatment-the-use-of-ataluren-in-the-effective-management-of-duchenne-muscular-dystrophy/
This emphasises the need for early genetic testing to identify the mutation, guide treatment and inform genetic counselling. Treatments for DMD ...
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https://touchneurology.com/neuromuscular-diseases/journal-articles/early-diagnosis-and-treatment-the-use-of-ataluren-in-the-effective-management-of-duchenne-muscular-dystrophy/
This emphasises the need for early genetic testing to identify the mutation, guide treatment and inform genetic counselling. Treatments for DMD ...
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14
In vivo genome editing improves muscle function in a mouse ...
https://www.science.org/doi/10.1126/science.aad5143
Because of the genetic nature of the disease, gene therapy is a promising option to treat DMD. However, the very large size of the dystrophin cDNA presents a ...
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https://www.science.org/doi/10.1126/science.aad5143
Because of the genetic nature of the disease, gene therapy is a promising option to treat DMD. However, the very large size of the dystrophin cDNA presents a ...
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15
2021 Duchenne Muscular Dystrophy Highlight - New Potential ...
https://cdmrp.health.mil/dmdrp/research_highlights/21McNally_Demonbreun_highlight.aspx
There is no cure for DMD, and patients typically do not survive past their 30s. Steroids are the primary treatment to slow DMD progression and ...
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https://cdmrp.health.mil/dmdrp/research_highlights/21McNally_Demonbreun_highlight.aspx
There is no cure for DMD, and patients typically do not survive past their 30s. Steroids are the primary treatment to slow DMD progression and ...
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16
Consensus on the diagnosis, treatment and follow-up of ...
https://www.sciencedirect.com/science/article/pii/S2173580818300476
Currently, DMD is mainly managed with multidisciplinary symptomatic treatment, with favourable results in terms of the progression of the disease. It is ...
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https://www.sciencedirect.com/science/article/pii/S2173580818300476
Currently, DMD is mainly managed with multidisciplinary symptomatic treatment, with favourable results in terms of the progression of the disease. It is ...
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17
About Duchenne Muscular Dystrophy
https://www.genome.gov/Genetic-Disorders/Duchenne-Muscular-Dystrophy
Treatment for Duchenne muscular dystrophy is aimed at the symptoms. Aggressive management of dilated cardiomyopathy with anti-congestive ...
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https://www.genome.gov/Genetic-Disorders/Duchenne-Muscular-Dystrophy
Treatment for Duchenne muscular dystrophy is aimed at the symptoms. Aggressive management of dilated cardiomyopathy with anti-congestive ...
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18
Current Translational Research and Murine Models For ...
https://content.iospress.com/articles/journal-of-neuromuscular-diseases/jnd150113
Currently, there is no cure for DMD. Since murine models are relatively easy to genetically manipulate, cost effective, and easily ...
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https://content.iospress.com/articles/journal-of-neuromuscular-diseases/jnd150113
Currently, there is no cure for DMD. Since murine models are relatively easy to genetically manipulate, cost effective, and easily ...
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19
Gene-Based Therapy for Duchenne Muscular Dystrophy (DMD)
https://www.aetna.com/cpb/medical/data/900_999/0911.html
The condition may occur in people who do not have a family history of DMD. There is no known cure for DMD. Treatment is targeted at controlling symptoms and ...
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https://www.aetna.com/cpb/medical/data/900_999/0911.html
The condition may occur in people who do not have a family history of DMD. There is no known cure for DMD. Treatment is targeted at controlling symptoms and ...
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20
Developments in reading frame restoring therapy approaches ...
https://www.tandfonline.com/doi/pdf/10.1080/14712598.2021.1832462
treatment of Duchenne muscular dystrophy and clinical trials are ... 2013;31. (7):397–405. 96. Ousterout DG, Perez-Pinera P, Thakore PI, ...
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https://www.tandfonline.com/doi/pdf/10.1080/14712598.2021.1832462
treatment of Duchenne muscular dystrophy and clinical trials are ... 2013;31. (7):397–405. 96. Ousterout DG, Perez-Pinera P, Thakore PI, ...
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21
Emerging Strategies in the Treatment of Duchenne Muscular ...
https://link.springer.com/article/10.1007/s13311-018-00687-z
Currently, there are 3 gene therapy programs for DMD that are actively enrolling. All utilize a muscle-specific promoter that is driving ...
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https://link.springer.com/article/10.1007/s13311-018-00687-z
Currently, there are 3 gene therapy programs for DMD that are actively enrolling. All utilize a muscle-specific promoter that is driving ...
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22
Empowering Muscle Stem Cells for the Treatment of ...
https://www.karger.com/Article/FullText/514305
Importantly, there remains no effective cure for DMD. DMD is caused by defective expression of the DMD gene, which encodes for dystrophin, a component of ...
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https://www.karger.com/Article/FullText/514305
Importantly, there remains no effective cure for DMD. DMD is caused by defective expression of the DMD gene, which encodes for dystrophin, a component of ...
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23
Long-term treatment with eteplirsen in nonambulatory patient...
https://journals.lww.com/md-journal/fulltext/2019/06280/long_term_treatment_with_eteplirsen_in.5.aspx
Duchenne muscular dystrophy (DMD) is a progressive, X-linked recessive, degenerative, universally fatal neuromuscular disease caused by lack of dystrophin that ...
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https://journals.lww.com/md-journal/fulltext/2019/06280/long_term_treatment_with_eteplirsen_in.5.aspx
Duchenne muscular dystrophy (DMD) is a progressive, X-linked recessive, degenerative, universally fatal neuromuscular disease caused by lack of dystrophin that ...
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24
DMD Treatment Candidate DYNE-251 Wins FDA Fast Track ...
https://musculardystrophynews.com/news/dmd-treatment-candidate-dyne-251-wins-fda-fast-track-status/
There is no known cure for DMD, but therapies that skip specific exons and enable the production of a smaller but functional form of dystrophin ...
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https://musculardystrophynews.com/news/dmd-treatment-candidate-dyne-251-wins-fda-fast-track-status/
There is no known cure for DMD, but therapies that skip specific exons and enable the production of a smaller but functional form of dystrophin ...
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25
Mutation-Based Therapy for Duchenne Muscular Dystrophy
https://www.ahajournals.org/doi/10.1161/CIRCULATIONAHA.117.028382
This treatment is directed at ≈10% to 15% of patients with DMD (≈1500 treatment-eligible individuals). The approval of eteplirsen is game ...
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https://www.ahajournals.org/doi/10.1161/CIRCULATIONAHA.117.028382
This treatment is directed at ≈10% to 15% of patients with DMD (≈1500 treatment-eligible individuals). The approval of eteplirsen is game ...
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26
Hope for a Cure for Duchenne Muscular Dystrophy
https://www.brainandlife.org/articles/hope-for-cure-duchenne-muscular-dystrophy
In the United Kingdom (UK), where Dr. Willis practices, trials for gene therapy and other investigative disease-modifying drugs have begun. Currently, ataluren ...
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https://www.brainandlife.org/articles/hope-for-cure-duchenne-muscular-dystrophy
In the United Kingdom (UK), where Dr. Willis practices, trials for gene therapy and other investigative disease-modifying drugs have begun. Currently, ataluren ...
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27
A Duchenne Muscular Dystrophy (DMD) Overview for ...
https://www.duchenne.com/hcp/duchenne-muscular-dystrophy-overview/what-dmd
Eteplirsen for the treatment of Duchenne muscular dystrophy. Ann Neurol. 2013;74:637-647. 2. Aartsma-Rus A, Ginjaar lB, Bushby K. The importance of genetic ...
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https://www.duchenne.com/hcp/duchenne-muscular-dystrophy-overview/what-dmd
Eteplirsen for the treatment of Duchenne muscular dystrophy. Ann Neurol. 2013;74:637-647. 2. Aartsma-Rus A, Ginjaar lB, Bushby K. The importance of genetic ...
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28
CRISPR technologies for the treatment of Duchenne muscular ...
https://www.cell.com/molecular-therapy-family/molecular-therapy/pdf/S1525-0016(21)00189-1.pdf
eteplirsen-treated DMD patients.11 All of the patients treated with ... Weerd, L., et al. (2013). Low dystrophin levels increase survival and improve muscle.
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https://www.cell.com/molecular-therapy-family/molecular-therapy/pdf/S1525-0016(21)00189-1.pdf
eteplirsen-treated DMD patients.11 All of the patients treated with ... Weerd, L., et al. (2013). Low dystrophin levels increase survival and improve muscle.
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29
What parents need to know about Duchenne muscular ...
https://www.youtube.com/watch?v=7EHdCQn6M4Y
Demystifying Medicine McMaster
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https://www.youtube.com/watch?v=7EHdCQn6M4Y
Demystifying Medicine McMaster
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30
The advances and challenges of Gene Therapy for Duchenne ...
https://www.heighpubs.org/jgmgt/jgmgt-aid1003.php
Since the discovery of the dystrophin gene (DMD gene) thirty years ago, several therapeutic approaches have been investigated to treat ...
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https://www.heighpubs.org/jgmgt/jgmgt-aid1003.php
Since the discovery of the dystrophin gene (DMD gene) thirty years ago, several therapeutic approaches have been investigated to treat ...
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31
Long-term benefits and adverse effects of intermittent versus ...
https://www.northstardmd.com/uploads/2/4/5/5/24555974/j_neurol_neurosurg_psychiatry-2013-ricotti-698-705.pdf
J Neurol Neurosurg Psychiatry 2013;84:698–705. doi:10.1136/jnnp-2012-303902. Neuromuscular ... dinal natural history data on boys with DMD treated and.
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https://www.northstardmd.com/uploads/2/4/5/5/24555974/j_neurol_neurosurg_psychiatry-2013-ricotti-698-705.pdf
J Neurol Neurosurg Psychiatry 2013;84:698–705. doi:10.1136/jnnp-2012-303902. Neuromuscular ... dinal natural history data on boys with DMD treated and.
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32
DMD-Ataluren (Translarna™), for Genetic Disorders
https://www.ptcbio.com/our-pipeline-areas-of-interest-ataluren/
Translarna is the first treatment approved for the underlying cause of DMD. The European Medicines Agency, or EMA, has designated ataluren as an orphan ...
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https://www.ptcbio.com/our-pipeline-areas-of-interest-ataluren/
Translarna is the first treatment approved for the underlying cause of DMD. The European Medicines Agency, or EMA, has designated ataluren as an orphan ...
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33
Repeated intravenous cardiosphere-derived cell therapy in ...
https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(22)00012-5/fulltext
Cardiosphere-derived cells (CDCs) ameliorate skeletal and cardiac muscle deterioration in experimental models of Duchenne muscular dystrophy ...
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https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(22)00012-5/fulltext
Cardiosphere-derived cells (CDCs) ameliorate skeletal and cardiac muscle deterioration in experimental models of Duchenne muscular dystrophy ...
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34
HTAQ Summer 2017 Duchenne Muscular Dystrophy New and ...
https://www.xcenda.com/insights/htaq-summer-2017-duchenne-muscular-dystrophy-new-and-revitalized-drugs-bring-hope-to-patients
DMD Treatment Landscape ... Unfortunately, there is no cure for DMD. Since patients with DMD are missing dystrophin—the essential structural ...
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https://www.xcenda.com/insights/htaq-summer-2017-duchenne-muscular-dystrophy-new-and-revitalized-drugs-bring-hope-to-patients
DMD Treatment Landscape ... Unfortunately, there is no cure for DMD. Since patients with DMD are missing dystrophin—the essential structural ...
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35
Strategies Learned from Duchenne Muscular Dystrophy
https://www.appliedclinicaltrialsonline.com/view/rare-disease-clinical-trials-strategies-learned-from-duchenne-muscular-dystrophy
As a result, standard clinical care guidelines for DMD are being ... as worthwhile relative to other experimental or approved treatments.
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https://www.appliedclinicaltrialsonline.com/view/rare-disease-clinical-trials-strategies-learned-from-duchenne-muscular-dystrophy
As a result, standard clinical care guidelines for DMD are being ... as worthwhile relative to other experimental or approved treatments.
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36
Duchenne Muscular Dystrophy - TREAT-NMD
https://treat-nmd.org/what-we-do/data-and-information/treat-nmd-diseases/duchenne-muscular-dystrophy/
It is caused by a fault in a gene called the dystrophin or DMD gene. A fault in this gene stops the body making a protein called dystrophin. This protein is ...
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https://treat-nmd.org/what-we-do/data-and-information/treat-nmd-diseases/duchenne-muscular-dystrophy/
It is caused by a fault in a gene called the dystrophin or DMD gene. A fault in this gene stops the body making a protein called dystrophin. This protein is ...
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37
"This Month in Muscular Dystrophy"
https://www.nationwidechildrens.org/research/areas-of-research/center-for-gene-therapy/muscular-dystrophy-podcasts
Dr. Jerry Mendell discusses phase 1/2a follistatin gene therapy trial for becker ... discusses IRES-Induced Dystrophin as a potential therapy for DMD.
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https://www.nationwidechildrens.org/research/areas-of-research/center-for-gene-therapy/muscular-dystrophy-podcasts
Dr. Jerry Mendell discusses phase 1/2a follistatin gene therapy trial for becker ... discusses IRES-Induced Dystrophin as a potential therapy for DMD.
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38
Gene therapies that restore dystrophin expression ... - UNC BME
https://www.bme.unc.edu/wp-content/uploads/sites/917/2018/07/Robinson-Hamm-Gersbach-2016-Hum-Genet-review-DMD-therapy.pdf
treatment of Duchenne muscular dystrophy. Jacqueline N. Robinson‑Hamm1,2 ... 2013), but they do not ultimately address the cause of DMD.
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https://www.bme.unc.edu/wp-content/uploads/sites/917/2018/07/Robinson-Hamm-Gersbach-2016-Hum-Genet-review-DMD-therapy.pdf
treatment of Duchenne muscular dystrophy. Jacqueline N. Robinson‑Hamm1,2 ... 2013), but they do not ultimately address the cause of DMD.
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39
Daily Steroids Safe and Slow Progression of Duchenne ...
https://www.urmc.rochester.edu/news/story/daily-steroids-safe-and-slow-progression-of-duchenne-muscular-dystrophy
“Corticosteroids are likely to remain the main treatment for DMD worldwide for the foreseeable future, so it is critical that we establish a ...
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https://www.urmc.rochester.edu/news/story/daily-steroids-safe-and-slow-progression-of-duchenne-muscular-dystrophy
“Corticosteroids are likely to remain the main treatment for DMD worldwide for the foreseeable future, so it is critical that we establish a ...
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40
Animal models of Duchenne muscular dystrophy
https://journals.biologists.com/Toolbox/DownloadCombinedArticleAndSupplmentPdf?resourceId=3670&multimediaId=1869267&pdfUrl=%2Fcob%2Fcontent_public%2Fjournal%2Fdmm%2F8%2F3%2F10.1242_dmm.018424%2F4%2F195.pdf
for DMD gene therapy (Fig. ... Konieczny et al., 2013; Mendell et al., 2012; Verhaart and Aartsma- ... to develop innovative therapies for treating DMD.
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https://journals.biologists.com/Toolbox/DownloadCombinedArticleAndSupplmentPdf?resourceId=3670&multimediaId=1869267&pdfUrl=%2Fcob%2Fcontent_public%2Fjournal%2Fdmm%2F8%2F3%2F10.1242_dmm.018424%2F4%2F195.pdf
for DMD gene therapy (Fig. ... Konieczny et al., 2013; Mendell et al., 2012; Verhaart and Aartsma- ... to develop innovative therapies for treating DMD.
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41
Duchenne muscular dystrophy: an historical treatment review
https://www.scielo.br/j/anp/a/3HwxGdn95vQ9DWY7JzCCgrS/
Despite these studies of side effects, both corticosteroid drugs (prednisone and deflazacort) remain the most important therapy for DMD patients, extending the ...
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https://www.scielo.br/j/anp/a/3HwxGdn95vQ9DWY7JzCCgrS/
Despite these studies of side effects, both corticosteroid drugs (prednisone and deflazacort) remain the most important therapy for DMD patients, extending the ...
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42
Micro-dystrophin and follistatin co-delivery restores muscle ...
https://academic.oup.com/hmg/article/22/24/4929/567402
Human Molecular Genetics, Volume 22, Issue 24, 15 December 2013, ... Gene delivery strategies to treat DMD have been under investigation for over a decade ...
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https://academic.oup.com/hmg/article/22/24/4929/567402
Human Molecular Genetics, Volume 22, Issue 24, 15 December 2013, ... Gene delivery strategies to treat DMD have been under investigation for over a decade ...
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43
Immune-mediated Mechanisms and Immunotherapy of ...
https://clinmedjournals.org/articles/jmdt/journal-of-musculoskeletal-disorders-and-treatment-jmdt-2-007.php?jid=jmdt
Immunotherapy has been developed to manage DMD, and glucocorticoids are used to treat DMD patients due to their anti-inflammatory functions. Furthermore, immune ...
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https://clinmedjournals.org/articles/jmdt/journal-of-musculoskeletal-disorders-and-treatment-jmdt-2-007.php?jid=jmdt
Immunotherapy has been developed to manage DMD, and glucocorticoids are used to treat DMD patients due to their anti-inflammatory functions. Furthermore, immune ...
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44
Long-term treatment with eteplirsen in nonambulatory patients ...
https://digitalcommons.wustl.edu/cgi/viewcontent.cgi?article=8996&context=open_access_pubs
Ann Neurol 2013;74:637–47. [3] McNeil DE, Davis C, Jillapalli D, et al. Duchenne muscular dystrophy: drug development and regulatory ...
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https://digitalcommons.wustl.edu/cgi/viewcontent.cgi?article=8996&context=open_access_pubs
Ann Neurol 2013;74:637–47. [3] McNeil DE, Davis C, Jillapalli D, et al. Duchenne muscular dystrophy: drug development and regulatory ...
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45
Duchenne muscular dystrophy hiPSC–derived myoblast drug ...
https://insight.jci.org/articles/view/134287
Following treatment of the cells with 1524 compounds from the Johns Hopkins Clinical Compound Library, compounds that enhanced myogenic fusion of DMD ...
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https://insight.jci.org/articles/view/134287
Following treatment of the cells with 1524 compounds from the Johns Hopkins Clinical Compound Library, compounds that enhanced myogenic fusion of DMD ...
→ Check Latest Keyword Rankings ←
46
Therapeutic Strategies for Duchenne Muscular Dystrophy
https://www.mdpi.com/2073-4425/11/8/837
Vector-mediated gene therapy delivers functional DMD gene to cells lacking dystrophin protein [41]. Another emerging strategy is CRISPR/Cas9-mediated gene ...
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https://www.mdpi.com/2073-4425/11/8/837
Vector-mediated gene therapy delivers functional DMD gene to cells lacking dystrophin protein [41]. Another emerging strategy is CRISPR/Cas9-mediated gene ...
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47
Search Orphan Drug Designations and Approvals - FDA
https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=403413
Date Designated: 08/16/2013. Orphan Designation: Treatment of Duchenne muscular dystrophy. Orphan Designation Status: Designated/Approved.
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https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=403413
Date Designated: 08/16/2013. Orphan Designation: Treatment of Duchenne muscular dystrophy. Orphan Designation Status: Designated/Approved.
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48
Guidance for Industry - Parent Project Muscular Dystrophy
https://www.parentprojectmd.org/wp-content/uploads/2021/07/2014_Community_Guidance.pdf
treatment of Duchenne muscular dystrophy (DMD) over the entire spectrum of the ... (McDonald 2013b) It is reliably obtained in younger DMD subjects99 and a ...
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https://www.parentprojectmd.org/wp-content/uploads/2021/07/2014_Community_Guidance.pdf
treatment of Duchenne muscular dystrophy (DMD) over the entire spectrum of the ... (McDonald 2013b) It is reliably obtained in younger DMD subjects99 and a ...
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49
Non-invasive assessment of skeletal muscle pathology and ...
https://www.oapublishinglondon.com/article/998
Non-invasive assessment of skeletal muscle pathology and treatment for Duchenne muscular dystrophy. OA Musculoskeletal Medicine 2013 Dec 20;1(4):33. Review ...
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https://www.oapublishinglondon.com/article/998
Non-invasive assessment of skeletal muscle pathology and treatment for Duchenne muscular dystrophy. OA Musculoskeletal Medicine 2013 Dec 20;1(4):33. Review ...
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50
Diagnosis and management of Duchenne muscular dystrophy ...
https://stacks.cdc.gov/view/cdc/52837/cdc_52837_DS2.pdf
the North Star Ambulatory Assessment in Duchenne muscular dystrophy. Dev Med Child Neurol. 2013 ... (2013) "Creatine for treating muscle disorders.".
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https://stacks.cdc.gov/view/cdc/52837/cdc_52837_DS2.pdf
the North Star Ambulatory Assessment in Duchenne muscular dystrophy. Dev Med Child Neurol. 2013 ... (2013) "Creatine for treating muscle disorders.".
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51
Toward the correction of muscular dystrophy by gene editing
https://www.pnas.org/doi/10.1073/pnas.2004840117
Duchenne muscular dystrophy (DMD) is an especially severe genetic ... up-regulation of the related gene utrophin, treatment with agents that ...
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https://www.pnas.org/doi/10.1073/pnas.2004840117
Duchenne muscular dystrophy (DMD) is an especially severe genetic ... up-regulation of the related gene utrophin, treatment with agents that ...
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52
Daily steroids safe and slows progression of duchenne ...
https://www.sciencedaily.com/releases/2022/05/220504135635.htm
"Corticosteroids are likely to remain the main treatment for DMD for the foreseeable future and worldwide so it is critical that we ...
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https://www.sciencedaily.com/releases/2022/05/220504135635.htm
"Corticosteroids are likely to remain the main treatment for DMD for the foreseeable future and worldwide so it is critical that we ...
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53
Emerging Drugs for Duchenne Muscular Dystrophy - CADTH
https://www.cadth.ca/emerging-drugs-duchenne-muscular-dystrophy
The mainstays of current therapy to treat DMD are corticosteroids and assistive devices. This bulletin focuses on new and emerging drugs for DMD with ...
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https://www.cadth.ca/emerging-drugs-duchenne-muscular-dystrophy
The mainstays of current therapy to treat DMD are corticosteroids and assistive devices. This bulletin focuses on new and emerging drugs for DMD with ...
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54
Muscular Dystrophy: Options for Complication Management
https://www.uspharmacist.com/article/muscular-dystrophy-options-for-complication-management
Pharmacologic Treatment. Clinical trials for MD are usually performed in patients with DMD, as it is the most common form. Sample sizes, however ...
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https://www.uspharmacist.com/article/muscular-dystrophy-options-for-complication-management
Pharmacologic Treatment. Clinical trials for MD are usually performed in patients with DMD, as it is the most common form. Sample sizes, however ...
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55
Current management of Duchenne muscular dystrophy in the ...
https://www.futuremedicine.com/doi/10.2217/nmt-2019-0002
In EU countries it is indicated for the treatment of DMD resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged ...
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https://www.futuremedicine.com/doi/10.2217/nmt-2019-0002
In EU countries it is indicated for the treatment of DMD resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged ...
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56
Longitudinal pulmonary function testing outcome measures in ...
https://www.nmd-journal.com/article/S0960-8966(18)30043-9/pdf
Abstract. We describe changes in pulmonary function measures across time in Duchenne muscular dystrophy patients treated with glucocorticoids.
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https://www.nmd-journal.com/article/S0960-8966(18)30043-9/pdf
Abstract. We describe changes in pulmonary function measures across time in Duchenne muscular dystrophy patients treated with glucocorticoids.
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57
Drug Discovery of Therapies for Duchenne Muscular Dystrophy
https://journals.sagepub.com/doi/10.1177/1087057115586535
Currently, there is no cure or disease-modifying therapy for DMD, and treatment is largely supportive and antisymptomatic. Glucocorticoids, ...
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https://journals.sagepub.com/doi/10.1177/1087057115586535
Currently, there is no cure or disease-modifying therapy for DMD, and treatment is largely supportive and antisymptomatic. Glucocorticoids, ...
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58
Duchenne Muscular Dystrophy - MDPI
https://mdpi-res.com/d_attachment/molecules/molecules-20-18168/article_deploy/molecules-20-18168.pdf?version=1444201297
Duchenne Muscular Dystrophy: From Diagnosis to Therapy ... two corticosteroids mainly used in DMD treatment are ... 2013, 23, 451–455.
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https://mdpi-res.com/d_attachment/molecules/molecules-20-18168/article_deploy/molecules-20-18168.pdf?version=1444201297
Duchenne Muscular Dystrophy: From Diagnosis to Therapy ... two corticosteroids mainly used in DMD treatment are ... 2013, 23, 451–455.
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59
Physical Therapy Guide to Muscular Dystrophies in Children
https://www.choosept.com/guide/physical-therapy-guide-muscular-dystrophies-in-children
According to one study, Duchenne muscular dystrophy is the most common form in children. An article in Nature Reviews states that DMD occurs in ...
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https://www.choosept.com/guide/physical-therapy-guide-muscular-dystrophies-in-children
According to one study, Duchenne muscular dystrophy is the most common form in children. An article in Nature Reviews states that DMD occurs in ...
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60
Oral muscles are progressively affected in Duchenne ...
https://mfm-nmd.org/upload-files/Oral_Musle_DMD.pdf
dystrophy: implications for dysphagia treatment ... Keywords Duchenne muscular dystrophy 4 Dysphagia 4 ... J Neurol (2013) 260:1295–1303.
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https://mfm-nmd.org/upload-files/Oral_Musle_DMD.pdf
dystrophy: implications for dysphagia treatment ... Keywords Duchenne muscular dystrophy 4 Dysphagia 4 ... J Neurol (2013) 260:1295–1303.
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61
Suitability of external controls for drug evaluation in Duchenne ...
https://n.neurology.org/content/95/10/e1381
The enrollment challenges stem from the rarity of DMD itself, the further rarity of certain treatment-amenable genetic subtypes, ...
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https://n.neurology.org/content/95/10/e1381
The enrollment challenges stem from the rarity of DMD itself, the further rarity of certain treatment-amenable genetic subtypes, ...
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62
Mediated DMD Exon Skipping in the mdx Mouse
https://escholarship.org/content/qt1td1d0v0/qt1td1d0v0_noSplash_838e913ebea346027863f068c722e74c.pdf
tion on DMD exon-23 skipping over long-term mdx treatment ... Alfano, L., Gomez, A.M., Lewis, S., Kota, J., et al.; Eteplirsen Study Group (2013).
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https://escholarship.org/content/qt1td1d0v0/qt1td1d0v0_noSplash_838e913ebea346027863f068c722e74c.pdf
tion on DMD exon-23 skipping over long-term mdx treatment ... Alfano, L., Gomez, A.M., Lewis, S., Kota, J., et al.; Eteplirsen Study Group (2013).
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63
Deflazacort, Eteplirsen, and Golodirsen for Duchenne ... - ICER
https://icer.org/wp-content/uploads/2020/10/ICER_DMD_Final_Scope_021119.pdf
with DMD usually progress to a loss of ambulation by age 10; treatment can ... Corticosteroids are the mainstay of therapy for DMD. ... 2013;61(9):948-954.
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https://icer.org/wp-content/uploads/2020/10/ICER_DMD_Final_Scope_021119.pdf
with DMD usually progress to a loss of ambulation by age 10; treatment can ... Corticosteroids are the mainstay of therapy for DMD. ... 2013;61(9):948-954.
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64
Diagnosis and management of Duchenne muscular dystrophy ...
https://childmuscleweakness.org/wp-content/uploads/2019/05/Duchenne_CareConsiderations_2018_Part3.pdf
muscular dystrophy (DMD) care considerations.1,2 ... Determine whether the patient is treated with chronic ... Neuromuscul Disord 2013;.
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https://childmuscleweakness.org/wp-content/uploads/2019/05/Duchenne_CareConsiderations_2018_Part3.pdf
muscular dystrophy (DMD) care considerations.1,2 ... Determine whether the patient is treated with chronic ... Neuromuscul Disord 2013;.
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65
Jerry R. Mendell, Ph.D. - Ohio State University
https://osuchildrensmusclegroup.org/mendell.shtml
Dr. Mendell is the first to perform gene therapy for DMD (March 2007) and also ... 2013;48:21-6. ... Duchenne muscular dystrophy: CRISPR/Cas9 treatment.
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https://osuchildrensmusclegroup.org/mendell.shtml
Dr. Mendell is the first to perform gene therapy for DMD (March 2007) and also ... 2013;48:21-6. ... Duchenne muscular dystrophy: CRISPR/Cas9 treatment.
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66
An alternative to corticosteroid for the treatment of Duchenne ...
https://www.worldmusclesociety.org/news/view/an-alternative-to-corticosteroid-for-the-treatment-of-duchenne-muscular-dystrophy
Duchenne Muscular dystrophy is characterized by progressive weakness ... in 2013 and joined the laboratory of Dr M. Carrie Miceli at UCLA, ...
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https://www.worldmusclesociety.org/news/view/an-alternative-to-corticosteroid-for-the-treatment-of-duchenne-muscular-dystrophy
Duchenne Muscular dystrophy is characterized by progressive weakness ... in 2013 and joined the laboratory of Dr M. Carrie Miceli at UCLA, ...
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67
Press Release - Investor Relations | Sarepta Therapeutics, Inc.
https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-signs-exclusive-global-collaboration-duke
... Technology to Develop New Treatments for Duchenne Muscular Dystrophy (DMD) ... Molecular Therapy 2013; Molecular Therapy 2015; ...
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https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-signs-exclusive-global-collaboration-duke
... Technology to Develop New Treatments for Duchenne Muscular Dystrophy (DMD) ... Molecular Therapy 2013; Molecular Therapy 2015; ...
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68
Muscular Dystrophy: Practice Essentials, Pathophysiology ...
https://emedicine.medscape.com/article/1259041-overview
Despite modern advances in gene therapy and molecular biology, MD remains incurable. With proper care and attention, patients have a better ...
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https://emedicine.medscape.com/article/1259041-overview
Despite modern advances in gene therapy and molecular biology, MD remains incurable. With proper care and attention, patients have a better ...
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69
ReveraGen BioPharma Inc Initiates VISION-DMD Phase 2a ...
https://www.reveragen.com/reveragen-biopharma-inc-initiates-vision-dmd-phase-2a-study-treatment-duchenne-muscular-dystrophy/
Enrollment has begun in the Phase 2a clinical trial of vamorolone (VBP15) in the treatment of boys with Duchenne muscular dystrophy (DMD).
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https://www.reveragen.com/reveragen-biopharma-inc-initiates-vision-dmd-phase-2a-study-treatment-duchenne-muscular-dystrophy/
Enrollment has begun in the Phase 2a clinical trial of vamorolone (VBP15) in the treatment of boys with Duchenne muscular dystrophy (DMD).
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70
News in DMD - Distrofia Muscular Progressiva
http://www.distrofiamuscular.net/news13.html
In Duchenne muscular dystrophy (DMD) and in the mdx mice model of DMD, ... 16 - (Pediatric Neurology, 2013) Corticosteroid Therapy for Duchenne Muscular ...
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http://www.distrofiamuscular.net/news13.html
In Duchenne muscular dystrophy (DMD) and in the mdx mice model of DMD, ... 16 - (Pediatric Neurology, 2013) Corticosteroid Therapy for Duchenne Muscular ...
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71
This Longhorn Has Been Running for a Cure for Duchenne ...
https://alcalde.texasexes.org/2022/09/longhorn-cure-for-duchenne-muscular-dystrophy/
... for Duchenne muscular dystrophy, a rare but devastating affliction. ... Life Member, took over in 2013, hosting the next three events.
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https://alcalde.texasexes.org/2022/09/longhorn-cure-for-duchenne-muscular-dystrophy/
... for Duchenne muscular dystrophy, a rare but devastating affliction. ... Life Member, took over in 2013, hosting the next three events.
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72
CD38‐NADase is a new major contributor to Duchenne ...
https://www.embopress.org/doi/10.15252/emmm.202012860
A pharmacological therapy to treat DMD has several advantages: It could ... in this genetic disease (Nigro et al, 1990; Kieny et al, 2013).
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https://www.embopress.org/doi/10.15252/emmm.202012860
A pharmacological therapy to treat DMD has several advantages: It could ... in this genetic disease (Nigro et al, 1990; Kieny et al, 2013).
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73
Duchenne muscular dystrophy - Wikipedia
https://en.wikipedia.org/wiki/Duchenne_muscular_dystrophy
Gene therapy, as a treatment, is in the early stages of study in humans. ... A small initial study using gene therapy has given some children improved muscle ...
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https://en.wikipedia.org/wiki/Duchenne_muscular_dystrophy
Gene therapy, as a treatment, is in the early stages of study in humans. ... A small initial study using gene therapy has given some children improved muscle ...
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74
The Problem with Clinical Trials in Duchenne—and What We ...
https://www.genengnews.com/topics/genome-editing/gene-therapy/the-problem-with-clinical-trials-in-duchenne-and-what-we-can-do-about-it/
The recent news that Sarepta Therapeutics is launching a Phase III trial of its leading gene therapy (SRP-9001) for Duchenne muscular dystrophy ...
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https://www.genengnews.com/topics/genome-editing/gene-therapy/the-problem-with-clinical-trials-in-duchenne-and-what-we-can-do-about-it/
The recent news that Sarepta Therapeutics is launching a Phase III trial of its leading gene therapy (SRP-9001) for Duchenne muscular dystrophy ...
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75
Duchenne muscular dystrophy
https://tidsskriftet.no/en/2014/08/duchenne-muscular-dystrophy
Treatment with glucocorticoids can slow disease progression and improve motor function in the short term. The treatment may cause side effects, ...
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https://tidsskriftet.no/en/2014/08/duchenne-muscular-dystrophy
Treatment with glucocorticoids can slow disease progression and improve motor function in the short term. The treatment may cause side effects, ...
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76
Understanding the Process of Fibrosis in Duchenne Muscular ...
https://www.hindawi.com/journals/bmri/2014/965631/
74, pp. 404–411, 2013. ... 2,. U. T. Ruegg, “Pharmacological prospects in the treatment of Duchenne muscular dystrophy,” Current Opinion in Neurology, vol. 26, pp ...
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https://www.hindawi.com/journals/bmri/2014/965631/
74, pp. 404–411, 2013. ... 2,. U. T. Ruegg, “Pharmacological prospects in the treatment of Duchenne muscular dystrophy,” Current Opinion in Neurology, vol. 26, pp ...
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77
Duchenne Muscular Dystrophy – Zebrafish Models
https://www.seattlechildrens.org/research/centers-programs/science-industry-partnerships/partnership-opportunities/duchenne-muscular-dystrophy/
Developing Treatments for Duchenne Muscular Dystrophy ... of Duchenne muscular dystrophy. PLOS Curr. 2013; ecurrents.md.8273cf41db10e2d15dd3ab827cb4b027 ...
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https://www.seattlechildrens.org/research/centers-programs/science-industry-partnerships/partnership-opportunities/duchenne-muscular-dystrophy/
Developing Treatments for Duchenne Muscular Dystrophy ... of Duchenne muscular dystrophy. PLOS Curr. 2013; ecurrents.md.8273cf41db10e2d15dd3ab827cb4b027 ...
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78
Cell Treatment Slows Disease in Muscular Dystrophy Patients
https://www.cedars-sinai.org/newsroom/cell-treatment-slows-disease-in-duchenne-muscular-dystrophy-patients/
The prognosis for patients with Duchenne muscular dystrophy is bleak. Most use a wheelchair by the time they are teenagers and don't typically ...
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https://www.cedars-sinai.org/newsroom/cell-treatment-slows-disease-in-duchenne-muscular-dystrophy-patients/
The prognosis for patients with Duchenne muscular dystrophy is bleak. Most use a wheelchair by the time they are teenagers and don't typically ...
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79
New study compares steroid regimens in boys with Duchenne ...
https://mmcneuro.wordpress.com/2013/05/18/new-study-compares-steroid-regimens-in-boys-with-duchenne-muscular-dystrophy/
Ongoing research is leading towards pre-symptomatic diagnosis of DMD, and there has been some progress in genetic therapy for affected boys ...
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https://mmcneuro.wordpress.com/2013/05/18/new-study-compares-steroid-regimens-in-boys-with-duchenne-muscular-dystrophy/
Ongoing research is leading towards pre-symptomatic diagnosis of DMD, and there has been some progress in genetic therapy for affected boys ...
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80
[PDF] Drug treatment of Duchenne muscular dystrophy
https://www.semanticscholar.org/paper/Drug-treatment-of-Duchenne-muscular-dystrophy%3A-and-BEYT%C3%8DA-Vry/08389d77388c00536e8288b3ec0f96d46f1659c2
› paper › Drug-treatment...
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https://www.semanticscholar.org/paper/Drug-treatment-of-Duchenne-muscular-dystrophy%3A-and-BEYT%C3%8DA-Vry/08389d77388c00536e8288b3ec0f96d46f1659c2
› paper › Drug-treatment...
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81
Therapeutic Approaches for Duchenne Muscular Dystrophy
https://www.researchgate.net/publication/349224798_Therapeutic_Approaches_for_Duchenne_Muscular_Dystrophy_Old_and_New
Gene replacement therapy, which is one of the focus points of this review, has emerged as the most promising potential treatment for DMD, ...
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https://www.researchgate.net/publication/349224798_Therapeutic_Approaches_for_Duchenne_Muscular_Dystrophy_Old_and_New
Gene replacement therapy, which is one of the focus points of this review, has emerged as the most promising potential treatment for DMD, ...
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82
Experimental gene therapy treatment for Duchenne muscular ...
https://medicalxpress.com/news/2013-02-experimental-gene-therapy-treatment-duchenne.html
Duchenne muscular dystrophy is caused by genetic mutations in the gene ... They expect results sometime in late 2013 or 2014 once all the ...
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https://medicalxpress.com/news/2013-02-experimental-gene-therapy-treatment-duchenne.html
Duchenne muscular dystrophy is caused by genetic mutations in the gene ... They expect results sometime in late 2013 or 2014 once all the ...
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83
Meet Dr. Mary Swift - Dallas Laser Dentistry
https://www.dallascosmeticdentist.us/meet-the-owner/
... dentist for comprehensive treatments, at Dallas Laser Dentistry in TX. ... Award (2013 – 15), and Living Magazine's Best Dallas Cosmetic Dentist (2014).
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https://www.dallascosmeticdentist.us/meet-the-owner/
... dentist for comprehensive treatments, at Dallas Laser Dentistry in TX. ... Award (2013 – 15), and Living Magazine's Best Dallas Cosmetic Dentist (2014).
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84
How to Treat a Cut in Your Mouth - Verywell Health
https://www.verywellhealth.com/how-to-treat-a-cut-inside-your-mouth-1059312
You should then rush to the dentist as soon as possible to seek treatment. This may involve dental splinting, socket repair, or tooth replacement.
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https://www.verywellhealth.com/how-to-treat-a-cut-inside-your-mouth-1059312
You should then rush to the dentist as soon as possible to seek treatment. This may involve dental splinting, socket repair, or tooth replacement.
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85
9 methods to get rid of toothache at night - Medical News Today
https://www.medicalnewstoday.com/articles/326133
It is important to seek immediate treatment from a dentist once a toothache lasts for more than a day or two. If dental pain is not treated straight away, it ...
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https://www.medicalnewstoday.com/articles/326133
It is important to seek immediate treatment from a dentist once a toothache lasts for more than a day or two. If dental pain is not treated straight away, it ...
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86
Florida Department of Health
https://www.floridahealth.gov/
... visited the expansion of the Coordinated Opioid Recovery (CORE) in Brevard County - a comprehensive network of addiction and opioid treatment.
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https://www.floridahealth.gov/
... visited the expansion of the Coordinated Opioid Recovery (CORE) in Brevard County - a comprehensive network of addiction and opioid treatment.
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87
Lhermitte's Sign in MS: What It Is & How To Treat It - WebMD
https://www.webmd.com/multiple-sclerosis/lhermittes-sign-what-is-how-treat
Daytona 500 winner Bayne revealed in fall 2013 he'd been diagnosed with multiple sclerosis. Doctors cleared the 22 year old to continue to compete in NASCAR, ...
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https://www.webmd.com/multiple-sclerosis/lhermittes-sign-what-is-how-treat
Daytona 500 winner Bayne revealed in fall 2013 he'd been diagnosed with multiple sclerosis. Doctors cleared the 22 year old to continue to compete in NASCAR, ...
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88
Dr turnipseed
https://indecenter.de/dr-turnipseed.html
A dentist is trained to diagnose, treat, and prevent diseases of the gums, ... 2013. edu The NeuroMedical Center is a leading evaluation and treatment ...
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https://indecenter.de/dr-turnipseed.html
A dentist is trained to diagnose, treat, and prevent diseases of the gums, ... 2013. edu The NeuroMedical Center is a leading evaluation and treatment ...
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89
Jaw Pain on One Side: 6 Causes, Relief Tips, When to See a ...
https://www.healthline.com/health/jaw-pain-one-side
Your dentist may recommend you wear the splint 24 hours a day, rather than just at night. Treatment may last from months to years.
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https://www.healthline.com/health/jaw-pain-one-side
Your dentist may recommend you wear the splint 24 hours a day, rather than just at night. Treatment may last from months to years.
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90
Duchenne muscular dystrophy - MedlinePlus
https://medlineplus.gov/ency/article/000705.htm
Causes · Symptoms · Exams and Tests · Treatment · Support Groups · Outlook (Prognosis) · Possible Complications · When to Contact a Medical ...
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https://medlineplus.gov/ency/article/000705.htm
Causes · Symptoms · Exams and Tests · Treatment · Support Groups · Outlook (Prognosis) · Possible Complications · When to Contact a Medical ...
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91
BNF (British National Formulary) | NICE
https://bnf.nice.org.uk/
Sep 28, 2022 —
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https://bnf.nice.org.uk/
Sep 28, 2022 —
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92
Copaxone uk
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