Category: News

News, Research

Progress in Motion: MDA’s 2019 Clinical & Scientific Conference Heralds New Directions for Neuromuscular Disease Treatment

MDA Staff

In April, MDA held its first ever combined Clinical & Scientific Conference, themed “Progress in Motion,” in Orlando Fla. Clinicians, scientists, policymakers, nonprofit, and industry leaders convened for a dynamic and informative five days in Orlando, Fla. With more than 1,200 attendees, 23 sessions, 136 presentations, and 300 posters, this was MDA’s largest conference ever. . . .

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News

Read the New MDA Manifesto

MDA Staff

Not long ago, supportive care and mobility aids were all medical science could offer people living with neuromuscular disease. But the treatment and care landscapes have begun to change dramatically. Recent breakthroughs in genetic medicine, along with other discoveries, are accelerating the development of new therapies, including some that not just address symptoms but also . . .

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News, Research, Uncategorized

Biogen Releases Letter to ALS Community

MDA Staff

Yesterday, Biogen released a letter to the ALS community regarding its experimental therapy, tofersen (formerly known as BIIB067), an antisense oligonucleotide being studied for the potential treatment of amyotrophic lateral sclerosis (ALS) in adults with a confirmed superoxide dismutase 1 (SOD1) genetic mutation, which is a subtype of familial ALS that makes up 2 percent . . .

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News, Research

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

Jeanene Swanson

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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News, Research

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

Jeanene Swanson

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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News, Partners, Research

Muscular Dystrophy Association Bridges Clinical and Academic Neuromuscular Research for the First Time at Annual Meeting

MDA Staff

With a laser focus on new horizons in neuromuscular research and care, the Muscular Dystrophy Association (MDA) is combining its clinical and scientific conferences for the first time, establishing a robust, state-of-the-art gathering for both clinicians and investigators from across the nation. Medical and scientific experts in neuromuscular disease from academia, private practice, government, and . . .

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News

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

Jeanene Swanson

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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Volunteering, News, Partners

Marriott International Associates Spend a Day Volunteering for MDA

MDA Staff

To kick off 2019 National Volunteer Month, the Muscular Dystrophy Association was honored to welcome Marriott International volunteers from all over the world into our Chicago offices on Friday, April 5, as part of Marriott’s Serve 360 event. Continuing its tradition of making its communities better places to live, work, and visit, Marriott International this . . .

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