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The first day of the 2023 MDA Clinical & Scientific Conference covered FDA approval, gene therapy, ultra-rare diseases, and more.

MDA Ambassador Leticia Tatum MDA Ambassador Leticia Tatum, 42, of Birmingham, Alabama was diagnosed with SMA at the age of two. She currently serves as the Vice President of Human…

As new treatments, cutting edge research, and a better understanding of genetic sequencing and mutations bring innovative change to the treatment landscape for neuromuscular disease, understanding your options and accessing…

MDA’s mission to fund groundbreaking research for neuromuscular disease treatment and provide families with the highest quality care would not be possible without the generous donation of time and resources…

Many Americans, including those living with a neuromuscular disease, rely on Medicaid to access vital medical care, especially during the COVID-19 pandemic. However, as the federal Public Health Emergency (PHE),…

Researchers at Alexion AZ Rare Disease are seeking adults living with generalized myasthenia gravis (gMG) to participate in a phase 2 clinical trial (ExpanD Study) to evaluate the safety and efficacy…

February 28 marked Rare Disease Day — an annual event calling attention to the 300 million people worldwide who live with one or more of the 7,000 illnesses classified as…

The 2023 MDA Clinical & Scientific Conference is just around the corner. With 32 sessions, 184 speakers, 61 exhibitors and patient advocacy organizations and 14 Industry Forums, this year’s conference…

On Feb. 28, the US Food and Drug Administration (FDA) granted approval to omaveloxolone (SKYCLARYS™) for the treatment of Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and…

By thinking about personal safety and learning about self-defense with a disability, you can feel more protected and confident outside your home.