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

Jeanene Swanson

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

Jeanene Swanson

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

Jeanene Swanson

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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Clinical Trial Alert: Amicus Therapeutics Seeks Teenage Participants for a Phase 3 Late-Onset Pompe Disease Study

MDA Staff

Researchers at Amicus Therapeutics are looking for adolescents (age 12-18) with late-onset Pompe disease (LOPD) to participate in a phase 3 study. The goal of the study is to evaluate the safety and efficacy of ATB200/AT2221, which researchers hope may improve muscle function and respiratory function compared to the standard of care. Participants will be . . .

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Casey’s General Stores Round Up for MDA

MDA Staff

This September, at Casey’s General Stores in 16 states across the Midwest, employees have been doing more than serving guests. They’ve been serving the MDA community, too, inviting store guests to round purchase totals up to the nearest whole dollar to support individuals and families impacted by neuromuscular disease. Now in its 13th year of . . .

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New MDA Educational Program Offers a 360-Degree View of ALS Care

MDA Staff

MDA’s new Medical Education department was established in response to the growing need for clinician education in neuromuscular diseases. We are pleased to present our first program highlighting amyotrophic lateral sclerosis (ALS): a PowerPoint slide deck. ALS is a motor neuron disease that leads to the widespread loss of muscle use. Patients diagnosed with ALS . . .

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2019 MDA Engage DMD Symposium Scheduled on Nov. 9

MDA Staff

At MDA, we believe in the power of research and the importance of building relationships among families, clinicians, and the scientists making discoveries to advance treatments and therapies. We invite individuals living with Duchenne muscular dystrophy (DMD), as well as their caregivers and loved ones, to attend the MDA Engage DMD Symposium taking place Nov. . . .

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2019 MDA Engage Charcot-Marie-Tooth Symposium Scheduled on Nov. 9

MDA Staff

At MDA, we believe in the power of research and the importance of building relationships among families, clinicians, and the scientists making discoveries to advance treatments and therapies. We invite individuals living with Charcot-Marie-Tooth disease (CMT), as well as caregivers and loved ones, to attend the MDA Engage CMT Symposium taking place Nov. 9 in . . .

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