Archives
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
Sarepta Therapeutics Announces Positive Results from the SRP-9001 Gene Therapy Trial to Treat DMD
On June 15, Sarepta Therapeutics announced positive results from its phase 1/2a study of SRP-9001 gene therapy to treat Duchenne muscular dystrophy (DMD). Data from four patients indicated that a…
On World FSHD Awareness Day 2020, MDA National Ambassador Tana Zwart Asks Us to Share Our Truths
“For any story of changing the world is always the story of many.” — Judith Heumann Tomorrow, June 20, is World FSHD Awareness Day, dedicated to facioscapulohumeral muscular dystrophy, a…
Momenta Pharmaceuticals Announces Positive Results from Phase 2 Vivacity-MG Study of Nipocalimab for Treatment of Generalized Myasthenia Gravis
On June 15, Momenta Pharmaceuticals announced positive results from its phase 2 Vivacity-MG trial assessing treatment with the company’s investigational therapeutic nipocalimab (M281) in patients with generalized myasthenia gravis (gMG).…
Hacking COVID: MDA Staff Edition
In April, MDA fielded a survey to ask its community how COVID-19 was impacting their lives. We heard your responses — anxiety, questions, hope — and wanted to know more.…
Tags: COVID-19, Mental Health, Staying Active