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The Expanding Therapeutic Landscape for Myasthenia Gravis
A Q&A with Gil I. Wolfe, MD
Expanding Access to Genetic Testing
MDA Care Centers bring no-cost genetic testing to people suspected of having a muscular dystrophy
Change for the Better
Advocacy for — and within — the neuromuscular disease community
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…
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…
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
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
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…
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
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