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Research Study Alert: Observational Study of Clinical and Electrophysiological Outcomes in Adults with CMT
Researchers at Ohio State University are seeking adults living with Charcot-Marie-Tooth disease type 1 (CMT-1) and type 2 (CMT-2) to participate in a one-month observational study (CMT Establish). This study is…
Clinical Trial Alert: Phase 3b Study of Oral Edaravone in Adults with ALS
Researchers at Mitsubishi Tanabe Pharma Development America, Inc. (MTPA) are seeking adults living with amyotrophic lateral sclerosis (ALS) to participate in a phase 3b clinical trial to evaluate the safety and…
Capricor Announces Positive Results from its Phase 2 Study of CAP-1002 to Treat DMD
On September 24, Capricor Therapeutics announced positive results at the World Muscle Society Virtual Congress from its phase 2 HOPE-2 trial of the investigational therapy CAP-1002 for treatment of people…
Improving Air Travel for People Living with a Disability
Broken wheelchairs. Inaccessible seating. Hard to navigate aircraft. For too many in the neuromuscular community, traveling by air is not accessible. We sat down with Paul Melmeyer, MDA’s VP of…
Clinical Trial Alert: Phase 3 Study of High Dose Eteplirsen in Boys with DMD
Researchers at Rare Disease Research, LLC are seeking boys between the ages of 7-years-old and 13-years-old who are living with Duchenne muscular dystrophy (DMD) to participate in the phase 3 MIS51ON…
Clinical Trial Alert: Phase 2 Study of Ataluren in Children with Nonsense Mutation DMD
Researchers at Rare Disease Research, LLC are seeking boys between the ages of 6 months and 2 years who are living with Duchenne muscular dystrophy caused by a nonsense mutation (nmDMD)…
Recognizing and Treating CMT in Kids
Like many middle-school kids, 11-year-old Callen, of Emmaus, Pennsylvania, has turned his mom, Jamie Moulthrop, into a chauffeur, she jokes. He participates in karate, baseball, hockey, and surfing, and he…
Tags: Healthcare, Parenting, Resources
Aging and Neuromuscular Disease Share Symptoms – and Solutions
With age often comes the maturity and grace to accept the world and oneself. That’s a good thing, because it takes every bit of that maturity to differentiate the aging…
Tags: Caregiving, Healthcare, Staying Active
Researchers Want to Know More About Oculopharyngeal Muscular Dystrophy
A Q&A with Anita Corbett, PhD
Tags: Research, Research Advances, Spotlight
One in a Million
While there are still challenges to researching therapies for ultra-rare neuromuscular diseases, many in the field see reasons for hope
Tags: Drug Development, Featured Content, Fundraising, Gene Therapy, Grants, Research, Research Advances