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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…
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…
Clinical Trial Alert: Mitsubishi Tanabe Pharma Seeks Participants for a Phase 3 ALS Study
Researchers at Mitsubishi Tanabe Pharma Development America are looking for participants with amyotrophic lateral sclerosis (ALS) to participate in a phase 3 study to evaluate the safety of oral edaravone…
Roche Genentech Announces Positive Results from Pivotal Study of Risdiplam in SMA Type 1
On Jan. 22, Genentech, a member of the Roche Group, announced positive results from the second part of its pivotal phase 2/3 FIREFISH clinical trial assessing risdiplam in infants age…
Clinical Trial Alert: LGMD2E Participants Sought for a Phase 1/2a Gene Delivery Study
Researchers at Sarepta Pharmaceuticals are looking for pediatric patients with limb-girdle muscular dystrophy type 2E (LGMD2E; aka beta-sarcoglycan deficiency) to participate in a phase 1/2a study. The goal of the…
MDA National Ambassadors Share the Everyday Impact of Neuromuscular Disease
In 1950, when the Muscular Dystrophy Association was founded, muscular dystrophy and neuromuscular disease weren’t well known or well understood. But they were, as today, affecting an entire community of…
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Clinical Trial Alert: Individuals with SMA Type 3 Sought for a Phase 2 Study
Researchers at Scholar Rock are looking for individuals with spinal muscular atrophy (SMA) type 3 to participate in a phase 2 study. The goal of the study is to evaluate…
MDA’s 2020 Advocacy Plan
It might only be a month into 2020, but MDA’s advocacy team and grassroots advocates are already making their voices heard. We expect the next 12 months to be crucial…
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