Archives
Clinical Trials During COVID-19: What We Need From the FDA
Over the course of the last several months, the US Food and Drug Administration (FDA) has undertaken many actions aimed at slowing the spread of the novel coronavirus and ensuring…
Tags: COVID-19, Clinical Trials
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
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
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
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…
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
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
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…
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 —…
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…