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Blog Post | Get Involved

This National Volunteer Week, Your Virtual Support of MDA Means Even More

Volunteers are a rich part of MDA’s 70-year history. Through volunteer committees, boards, community events, and Summer Camps where kids build confidence and independence, they are tightly woven into MDA’s…

Tags: Fundraising, Let's Play, Summer Camp, Volunteers


Blog Post | Advocacy

MDA Advocacy in These Uncertain Times

We know families across the country, especially those in the neuromuscular disease (NMD) community, are concerned about the spread of coronavirus and its associated disease, COVID-19, and the impact on…

Tags: Accessible Air Travel, Advocacy Updates, COVID-19, Drug Development, Employment, Healthcare, Insurance


Blog Post | News, Science + Research

Acceleron Pharma Announces Discontinuation of Clinical Trials Program for CMT

On March 9, Acceleron Pharma, Inc., announced that a phase 2 clinical trial of its therapy under development, ACE-083, for the treatment of Charcot-Marie-Tooth disease (CMT) did not show statistically significant…

Tags: Clinical Trials


Blog Post | News, Science + Research

NS Pharma Launches Expanded Access Program for Viltolarsen

On March 9, NS Pharma Inc. (a wholly owned US subsidiary of Nippon Shinyaku Co. Ltd.) announced that it has launched an Expanded Access Program in the United States for…

Tags: Clinical Trials, Drug Development, Exon Skipping, Research


Blog Post | Get Involved

What’s New for MDA Muscle Walk in 2020

Since 2011, MDA Muscle Walks have been gathering individuals living with muscular dystrophy, ALS, and related neuromuscular diseases with their families, friends, and communities across America. Together, they’ve raised awareness…

Tags: Fundraising, Muscle Walk


Blog Post | Health, Science + Research

Simply Stated: What is Inclusion-Body Myositis?

Myopathy is a disease condition where muscle fibers do not function properly, resulting in muscular weakness. Inclusion-body myositis (IBM) is a type of inflammatory myopathy that is characterized by inflammation,…

Tags: Research, Simply Stated


Blog Post | News, Science + Research

FDA Accepts NS Pharma’s NDA for Viltolarsen for the Treatment of DMD Amenable to Exon 53 Skipping

On Feb. 7, NS Pharma Inc. (a wholly owned US subsidiary of Nippon Shinyaku Co. Ltd.), announced that the US Food and Drug Administration (FDA) accepted its New Drug Application…

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


Blog Post | News, Science + Research

MDA’s New Chief Research Officer Sharon Hesterlee Shares Vision for the Future

Last year, Sharon Hesterlee, PhD, took the helm as executive vice president, chief research officer, at MDA. With her unique, decades-spanning career — which began, in fact, at MDA —…

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


Blog Post | News, Science + Research

Clinical Trial Alert: Expansion Therapeutics Seeks Participants for a Phase 1 DM1 Study

Researchers at Expansion Therapeutics are looking for adults with myotonic dystrophy type 1 (DM1) to participate in a phase 1 study. The goal of the study is to evaluate the…

Tags: Clnical Trial Alert, Drug Development, Research, Research Advances


Blog Post | News, Science + Research

Clinical Trial Alert: LGMD2I Participants Sought for a Phase 3 Study

Researchers at PTC Therapeutics are looking for individuals with limb-girdle muscular dystrophy type 2I (LGMD2I) to participate in a phase 3 study. The goal of the study is to evaluate…

Tags: Clnical Trial Alert, Drug Development, Research, Research Advances