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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…
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…
Smashing Stereotypes
Jessica Hetzel faced a long but rewarding journey to join Greek life on her college campus. Making sorority life more inclusive, one chapter at a time
MDA’s 2020 Advocacy Agenda Update
Even during the COVID-19 pandemic, MDA continues to make sure lawmakers hear the voice of the neuromuscular disease community
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