MDA Care Centers Combine Expert Care with Research, Clinical Trials, and Advanced Technology

At more than 150 of the leading healthcare institutions across the US, MDA Care Centers offer top clinical care to individuals living with muscular dystrophy, ALS, and other neuromuscular diseases. Providing access to this expert multidisciplinary care, however, is just one way that Care Centers serve the neuromuscular community. MDA Care Centers are conducting clinical . . .

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World Muscle Society Roundup: Clinical Trial Results of Treatments in Development for DMD, XLMTM, SMA, and Pompe Disease

Several biotech and pharma companies presented interim results at the 24th Annual Congress of the World Muscle Society (WMS) held earlier this month in Copenhagen, Denmark. This roundup covers a few of these updates. Catabasis Pharmaceuticals presented positive results from its phase 1/2 MoveDMD trial and open-label extension with edasalonexent in boys with Duchenne muscular . . .

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Reata Pharmaceuticals Announces Positive Results from Pivotal Study of Omaveloxolone in FA

Texas-based Reata Pharmaceuticals announced positive results of its pivotal phase 2 clinical trial (MOXIe) of omaveloxolone in patients with Friedreich’s ataxia (FA). Patients treated with omaveloxolone showed a statistically significant improvement in the modified Friedreich’s Ataxia Rating Scale mFARS (a scale used to measure neurological function) after 48 weeks of treatment compared to placebo, and . . .

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Capricor Therapeutics Announces Positive Interim Results from Phase 2 Study of CAP-1002 to Treat DMD

In July of this year, Capricor Therapeutics announced positive interim results from its phase 2 (HOPE-2) clinical trial evaluating CAP-1002, the company’s lead investigational therapy for Duchenne muscular dystrophy (DMD), to treat boys and young men in advanced stages of the disease. In that analysis, meaningful improvements in mid-level upper limb and hand function were . . .

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MDA Grants Charles Thornton More Than $1 Million in New Funding to Continue Supporting the Myotonic Dystrophy Clinical Research Network

Charles Thornton, MD, professor of Neurology at the University of Rochester, was awarded an MDA clinical research network grant (CRNG) totaling $1,118,673 over three years to continue to lead the development of the Myotonic Dystrophy Clinical Research Network, which comprises six medical centers specializing in research and clinical care of myotonic dystrophy (DM) types 1 . . .

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Five Questions with DMD Researcher Matthew Wood

Matthew Wood, PhD, professor of Neuroscience at the University of Oxford and director of both the Oxford Rare Disease Center and the MDUK Oxford Neuromuscular Center, was awarded an MDA research grant totaling $210,000 over two years to optimize the delivery of genetic therapies such as oligonucleotides to tissues for treating Duchenne muscular dystrophy (DMD). . . .

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ReveraGen Presents Positive Interim Results from Phase 2 Extension Study of Vamorolone to Treat DMD in Young Boys

At the World Muscle Society annual meeting held earlier this month in Copenhagen, Denmark, ReveraGen announced positive results from its ongoing phase 2 clinical trial of vamorolone for treating Duchenne muscular dystrophy (DMD) in boys. Building upon previously reported positive results, CEO Dr. Eric Hoffman presented data gathered after 18 months of treatment in the . . .

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NS Pharma Submits New Drug Application to FDA for Viltolarsen to Treat DMD Amenable to Exon 53 Skipping

On Oct. 2, NS Pharma Inc. (a wholly-owned US subsidiary of Nippon Shinyaku Co. Ltd.) submitted a rolling New Drug Application (NDA) to the US Food and Drug Administration (FDA) for its exon-skipping therapy under development, viltolarsen (previously NS-065/NCNP-01), to treat Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. On Sept. 26, Nippon Shinyaku . . .

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Five Questions with LGMD Researcher Martin Childers

Martin Childers, PhD, DO, chief medical officer at Asklepios BioPharmaceutical Inc. in North Carolina, was awarded an MDA research grant totaling $192,500 over one year to perform pre-clinical studies using an adeno-associated virus (AAV) to deliver a gene therapy for limb-girdle muscular dystrophy type 2I (LGMD2I). Limb-girdle muscular dystrophies are a diverse group of disorders . . .

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