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 . . .

Read More

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 . . .

Read More

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 . . .

Read More

MDA Wins 2019 Sonia Skarlatos Public Service Award

We are pleased to announce that on May 1, 2019, the American Society for Gene and Cell Therapy awarded MDA the Sonia Skarlatos Public Service Award for 2019. Named for its inaugural co-recipient and tireless gene therapy advocate, the ASGCT’s Sonia Skarlatos Public Service Award recognizes a person or group that has consistently fostered and . . .

Read More

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 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. . . .

Read More

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. 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 . . .

Read More

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 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 . . .

Read More

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 . . .

Read More