Strongly

An expression of the live unlimited spirit, stories and voices of the MDA community

News, Research

Research Study Alert: Researchers at Virginia Commonwealth University Seek Participants for a LGMD2I Natural History Study

MDA Staff

Researchers at Virginia Commonwealth University are seeking participants living with limb-girdle muscular dystrophy type 2I (LGMD2I) to participate in a natural history study. This study aims to better understand disease course so researchers will be able to determine clinically meaningful outcome measures for use in future clinical trials, including the utility of dystroglycan as a biomarker. This two-year . . .

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News, Research

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

Jeanene Swanson

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 (NDA) seeking approval under Priority Review for viltolarsen, an investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. Priority . . .

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News, Research

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

Jeanene Swanson

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 — in the pharmaceutical, biotechnology, and nonprofit sectors, her appointment is certain to advance not only MDA’s mission but also the research and development of genetic . . .

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News, Research

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

MDA Staff

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 the efficacy of deflazacort (Emflaza), which researchers hope may reduce inflammation in muscles and potentially lead to improved muscle strength and motor function. Participants will . . .

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Research

Clinical Trial Alert: Mitsubishi Tanabe Pharma Seeks Participants for a Phase 3 ALS Study

MDA Staff

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 (Radicava). Intravenous edaravone is an FDA-approved treatment for ALS shown to slow the decline of physical function. Edaravone is thought to act as a free . . .

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MDA Ambassadors, News, Living with Muscular Dystrophy

MDA National Ambassadors Share the Everyday Impact of Neuromuscular Disease

Lindsey Baker

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 kids and adults. And so in 1952, MDA made a move to introduce this community of individuals and families to the world. MDA’s National Ambassador . . .

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Advocacy

MDA’s 2020 Advocacy Plan

Mark Fisher

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 on Capitol Hill, and we’ll be working tirelessly in Washington, DC, to transform the lives of people living with neuromuscular disorders. Here’s a sneak peek . . .

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