Do You Know ALS? Read About the Latest Research.

It’s a hard fact that only three medications have been approved to treat amyotrophic lateral sclerosis (ALS) in nearly 25 years. Fortunately, research in ALS has exploded in the past decade, and new technologies have enabled the development of gene-targeting therapies such as gene replacement, gene silencing, and gene editing. MDA has always been committed . . .

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2019 MDA Engage ALS Symposium Scheduled in Boston on May 18

At MDA, we believe in the power of research and the importance of building relationships among families, clinicians, and the scientists making discoveries to advance treatments and therapies. We invite individuals living with a neuromuscular disease, as well as caregivers and loved ones, to attend MDA Engage symposia taking place across the country. MDA Engage . . .

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FDA Approves Jacobus Pharmaceuticals’ Ruzurgi for Treatment of Children with Lambert-Eaton Myasthenic Syndrome

On May 6, the U.S. Food and Drug Administration (FDA) announced the approval of Ruzurgi (amifampridine) for the treatment of children with Lambert-Eaton myasthenic syndrome (LEMS) who are between 6 and 17 years of age. This is the second FDA-approved treatment for LEMS and the first approved treatment for pediatric LEMS patients. Ruzurgi is an oral potassium . . .

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2019 MDA Engage LGMD Symposium Scheduled in Richmond on May 18

At MDA, we believe in the power of research and the importance of building relationships among families, clinicians, and the scientists making discoveries to advance treatments and therapies. We invite individuals living with a neuromuscular disease, as well as caregivers and loved ones, to attend MDA Engage symposia taking place across the country. MDA Engage . . .

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Mouse Study Finds Link Between Key Mitochondrial Protein and CMT2A, Making It a Possible Therapeutic Target

In a study conducted in mice, scientists led by an MDA-funded researcher found that increasing the amount of a certain mitochondrial membrane protein, mitofusin-1 (MFN1), lessened symptoms of Charcot-Marie-Tooth disease (CMT) type 2A. The findings suggest that it may be possible to treat CMT2A using gene-replacement therapies that deliver functional copies of missing or mutated . . .

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MDA Partners with the Broad Institute on Rare Genomes Project for Limb-Girdle Muscular Dystrophy

The Muscular Dystrophy Association (MDA) is pleased to announce a collaboration with the Broad Institute of MIT and Harvard, which aims to bring genetic diagnoses to more individuals living with limb-girdle muscle weakness. The goal of the LGMD Rare Genomes Project is to bring the most advanced genetic techniques, including whole-genome sequencing and genetic variation . . .

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Audentes Therapeutics Announces Plans to Develop Gene-Targeted Therapies for Duchenne Muscular Dystrophy and Myotonic Dystrophy Type 1

On April 8, Audentes Therapeutics announced that it will expand its gene-targeted therapy platform to include developing experimental drugs for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). The company has entered into a licensing agreement with Nationwide Children’s Hospital in Ohio and will collaborate with leading researchers in the field, Kevin M. Flanigan, . . .

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Muscular Dystrophy Association Awards Eight Grants Totaling More Than $2 Million for ALS Research

Today, the Muscular Dystrophy Association (MDA) announced the awarding of eight new grants totaling more than $2 million toward research focused on amyotrophic lateral sclerosis (ALS), a disease in which muscles become weak and eventually nonfunctional. Since its inception, MDA has invested more than $165 million in ALS research, and in the last five years . . .

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2019 MDA Engage SMA Symposium Scheduled in Orlando on April 12

At MDA, we believe in the power of research and the importance of building relationships among families, clinicians, and the scientists making discoveries to advance treatments and therapies. We invite individuals living with a neuromuscular disease, as well as caregivers and loved ones, to attend MDA Engage symposia taking place across the country. MDA Engage . . .

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Sarepta Therapeutics Announces Positive Interim Results from Study of Casimersen to Treat DMD Amenable to Exon 45 Skipping

On Mar. 28, Sarepta Therapeutics announced encouraging interim results from a phase 3 clinical trial that suggest the experimental drug casimersen potentially may be effective as a treatment for Duchenne muscular dystrophy (DMD). Earlier this year, the U.S. Food and Drug Administration (FDA) accepted Sarepta Therapeutics’ New Drug Application (NDA) seeking approval under Priority Review for . . .

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