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Blog Post | Science + Research

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…

Tags: Clinical Trials, Drug Development, Gene Therapy, Research, Research Advances


Blog Post | Inclusion, Independence, Lifestyle, Personal Stories

On World FSHD Awareness Day 2020, MDA National Ambassador Tana Zwart Asks Us to Share Our Truths

β€œFor any story of changing the world is always the story of many.” β€” Judith Heumann Tomorrow, June 20, is World FSHD Awareness Day, dedicated to facioscapulohumeral muscular dystrophy, a…

Tags: Accessible Air Travel, Ambassadors, Education, Employment


Blog Post | Science + Research

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).…

Tags: Clinical Trials, Drug Development, Research, Research Advances


Blog Post | Lifestyle, Personal Stories

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 β€” anxiety, questions, hope β€” and wanted to know more.…

Tags: COVID-19, Mental Health, Staying Active


Blog Post | Lifestyle, Personal Stories

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 β€” anxiety, questions, hope β€” and wanted to know more.…

Tags: Ambassadors, COVID-19, Mental Health


Blog Post | News, Research

Five Questions With CMT Researcher Stephan ZΓΌchner

Stephan ZΓΌchner, 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


Blog Post | News, Research

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


Blog Post | Independence, Lifestyle

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 β€” anxiety, questions, hope β€” and wanted to know more.…

Tags: Ambassadors, COVID-19, Mental Health


Blog Post | Independence, Lifestyle

Hacking COVID: Voices from the Community, Part 3

In April, MDA fielded a survey to ask its community how COVID-19 was impacting their lives. We heard your responses β€” anxiety, questions, hope β€” and wanted to know more.…

Tags: COVID-19, Mental Health


Blog Post | News, Research

ReveraGen BioPharma Announces Completion of Vamorolone Study in 41 DMD Patients

On June 2, ReveraGen BioPharma announced the completion of two-and-a-half years of vamorolone treatment in 41 boys with Duchenne muscular dystrophy (DMD). The long-term data from this study indicated that…

Tags: Clinical Trials, Research, Research Advances