Archives
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
Hacking COVID: MDA Ambassador Edition, Part 2
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: Ambassadors, COVID-19, Mental Health
Five Questions With CMT Researcher Stephan Züchner
Stephan Züchner, MD, PhD, professor for Human Genetics and Neurology, Chair of the Human Genetics Department, and codirector of the John P. Hussman Institute for Human Genomics at the University…
Tags: Research, Research Advances
Sarepta Therapeutics Announces Positive Results from the SRP-9003 Gene Therapy Trial to Treat LGMD2E
On June 8, Sarepta Therapeutics announced positive results from its first study in human patients of SRP-9003, a gene therapy designed to treat limb-girdle muscular dystrophy (LGMD) type 2E. Data…
Tags: Clinical Trials, Research, Research Advances
Hacking COVID: MDA Ambassador Edition, Part 1
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: Ambassadors, COVID-19, Mental Health