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Featured Article | Science + Research

The Expanding Therapeutic Landscape for Myasthenia Gravis

A Q&A with Gil I. Wolfe, MD

Tags: Drug Development, Research, Research Advances, Spotlight


Featured Article | Health, Science + Research

Expanding Access to Genetic Testing

MDA Care Centers bring no-cost genetic testing to people suspected of having a muscular dystrophy

Tags: Clinical Trials, Genetic Testing, Healthcare, MDA Care Centers


Featured Article | Advocacy, Inclusion, Independence

Change for the Better

Advocacy for — and within — the neuromuscular disease community

Tags: Accessible Air Travel, Advocacy Updates, COVID-19, Summer Camp


Blog Post | News, Research

FDA Approves NS Pharma’s Viltepso for Treatment of DMD Amenable to Exon 53 Skipping

Today, the US Food and Drug Administration (FDA) granted accelerated approval to viltolarsen (Viltepso) for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. It is…

Tags: Clinical Trials, Drug Approval, Exon Skipping


Blog Post | News, Research

FDA Approves Genentech’s Evrysdi for Treatment of SMA in Pediatric and Adult Patients

On Aug. 7, the US Food and Drug Administration (FDA) granted approval of risdiplam (Evrysdi) for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. It is the third…

Tags: Clinical Trials, Drug Approval, Drug Development


Blog Post | Independence, Lifestyle, Personal Stories

Creating a Network: How Hiring PCAs Gave Me Confidence and Independence, In College and Beyond

Josh Cueter, 22, from Troy, Michigan, is a 2020 graduate from Michigan State University.  At school he was a board member of the MSU Adaptive Sports & Recreation Club as…

Tags: College, Education, Personal Care Attendants, Staying Active, Summer Camp, Year of Independence, Young Adults


Blog Post | Get Involved, Personal Stories

A Q&A With MDA Let’s Play Community Manager Paul Scherer

In the gaming world, Paul Scherer goes by Puzzle. And as community manager of MDA Let’s Play, our dedicated community gaming and fundraising platform, he anticipates technical issues and seeks…

Tags: Innovation, Let's Play, Relationships, Staying Active


Blog Post | News, Research

Biogen Announces Positive Results from Phase 1/2 Study of Tofersen to Treat a Genetic Form of ALS

On July 8, Biogen announced positive results from its phase 1/2 clinical trial of the investigational therapy tofersen (BIIB067) for treatment of amyotrophic lateral sclerosis (ALS) caused by mutation of…

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


Blog Post | News, Science + Research

How MDA is Supporting LGMD Research and Care in 2020

On July 18, MDA is hosting a free virtual Engage Limb-Girdle Muscular Dystrophy Symposium, a half-day educational event featuring key experts who will speak on topics including the latest in…

Tags: Clinical Trials, Drug Development, Gene Therapy, MDA Care Centers, MOVR, Research, Research Advances


Blog Post | Health, Lifestyle

What the COVID-19 Pandemic Can Teach Us About the Isolation Those Living With Neuromuscular Disease Experience Every Day

In March 2020, MDA asked our advocates how the novel coronavirus pandemic was impacting their lives. New Yorker Robert Paulson shared his story — and the universal truth about how…

Tags: COVID-19