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National Ambassador Tana Zwart’s Take on MDA’s 2019 Clinical & Scientific Conference
Tana Zwart When getting acquainted with something new, you can toe-test or you can jump. As for my newly appointed MDA National Ambassador role, attending the inaugural MDA Clinical &…
Progress in Motion: MDA’s 2019 Clinical & Scientific Conference Heralds New Directions for Neuromuscular Disease Treatment
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…
Tags: Clinical Trials, Gene Therapy, Healthcare, Innovation, MDA Clinical and Scientific Conference, Newborn Screening, Research, Technology, Transitions
Read the New MDA Manifesto
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.…
Tags: Innovation
Biogen Releases Letter to ALS Community
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…
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…
Tags: Gene Therapy, Grants, Research
Clinical Trial Alert: PTC Therapeutics Seeks Participants for a Phase 3 DMD Study
Researchers at PTC Therapeutics are looking for participants with Duchenne muscular dystrophy (DMD) to participate in a Phase 3 study. The goal of the study is to characterize the long-term…
Introducing New MDA National Ambassador Tana Zwart, and Checking in With Ambassadors Faith Fortenberry and Justin Moy
Since the early 1950s, not long after the Muscular Dystrophy Association’s formation, America’s young people living with muscular dystrophy and related neuromuscular diseases have stepped forward to share their stories,…
Tags: Ambassadors, Community, Summer Camp, Young Adults
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…
Tags: Genetic Testing, Grants, Research
Muscular Dystrophy Association Bridges Clinical and Academic Neuromuscular Research for the First Time at Annual Meeting
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…
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).…