Strongly


An expression of the live unlimited spirit, stories and voices of the MDA community

Reporting Back on MDA’s First-Ever Virtual Hill Day

When the COVID-19 pandemic made meeting in person impossible, MDA’s Advocacy program needed to shift from in-person to virtual meetings with lawmakers. In early August, volunteers met with their lawmakers virtually — for the first time — to urge elected officials in Washington, DC, to end the process of surprise medical billing, which can be . . .

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FDA Accepts Sarepta Therapeutics’ New Drug Application for DMD Treatment Casimersen

On Aug. 25, Sarepta Therapeutics announced that the US Food and Drug Administration (FDA) accepted the company’s New Drug Application (NDA) seeking accelerated approval for casimersen (SRP-4045), an investigational therapy for Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping. The FDA has granted Sarepta’s application Priority Review Status, meaning that a decision is expected . . .

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Who’s On Your MDA Care Center Team?: Pediatric Social Workers

At more than 150 MDA Care Centers nationwide, kids and adults living with neuromuscular disease (NMD) have access to multidisciplinary care teams made up of different specialists who can coordinate in one place on an appointment day to optimize a complete care plan. Included on these teams are allied health professionals and social workers who . . .

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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 the third exon-skipping, disease-modifying drug to treat DMD, the most common childhood form of muscular dystrophy. The drug is administered by intravenous infusion. Viltepso will . . .

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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 well as MSU’s Council of Students with Disabilities.  After a semester internship with the Equal Employment Opportunity Commission in Washington, DC, Josh is currently working . . .

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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 the superoxide dismutase 1 gene (SOD1). The primary endpoints of the study — assessment of safety, tolerability, and drug dynamics in the body — demonstrated . . .

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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 genetics and research in limb-girdle muscular dystrophy (LGMD). MDA has a longstanding commitment to finding treatments for the more than 30 subtypes of LGMD; since . . .

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