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Blog Post | Science + Research

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…

Tags: Clinical Trial Alert, Clinical Trials, Research


Blog Post | Science + Research

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…

Tags: Clinical Trial Alert, Clinical Trials, Drug Development, Research


Blog Post | News, Research

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…

Tags: Clinical Trials, Drug Development, Research, Research Advances


Blog Post | Advocacy, Independence

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…

Tags: Accessible Air Travel, Advocacy Updates, Travel


Blog Post | Science + Research

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…

Tags: Clinical Trial Alert, Clinical Trials, Drug Development


Blog Post | Science + Research

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)…

Tags: Clinical Trial Alert, Clinical Trials, Drug Development


Blog Post | Health, Science + Research

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


Blog Post | Health

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


Featured Article | Science + Research

Researchers Want to Know More About Oculopharyngeal Muscular Dystrophy

A Q&A with Anita Corbett, PhD

Tags: Research, Research Advances, Spotlight


Featured Article | Health, Science + Research

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