Drug companies are developing more therapies for rare disease patients — and doing it faster — than ever before. But when it comes to enrolling minorities in clinical trials, the US track record is lagging behind. That subject was the focus of a March 15 session during the 2022 MDA Clinical & Scientific Conference in . . .
On April 5, an international research team, headed by Michela Guglieri at Newcastle University and Robert Griggs at the University of Rochester Medical Center, announced positive results at the American Academy of Neurology (AAN meeting) in Seattle, WA from the phase 3 FOR DMD study of corticosteroid dosing regimens for treatment of people with Duchenne . . .
For over 70 years, MDA has led the way as the #1 Voluntary Health Organization in the U.S. for people living with neuromuscular diseases. We build on our legacy of innovation in research, care, and fundraising by celebrating National Volunteer Month, to thank our volunteers who work tirelessly to support MDA’s mission and the families . . .
As the Muscular Dystrophy Association (MDA) continues our commitment to empowering people living with neuromuscular disease, we are excited to present our 2022 blog series: “Quest for Success”. Success looks different to everyone and this monthly blog details the different paths that individuals with neuromuscular disease have taken to reach their potential and the steps . . .
Myotonic dystrophy (DM) is a type of muscular dystrophy that affects about 1 in 8,000 people worldwide. The disease is known by several names, including Steinert disease, after the doctor that first described it, and “DM” in reference to its Greek name, dystrophia myotonica. Similar to other forms of muscular dystrophy, DM is characterized by . . .
When he was 15 years old, Elijah Stacy had a dream: to end Duchenne muscular dystrophy (DMD), the disease that affects him and thousands of other people. So, he decided to start a nonprofit called Destroy Duchenne to support advancing gene-editing and gene-therapy technologies to find a cure for DMD. To achieve his ambitious goal, . . .
To commemorate the 40th Birthday of MDA Shamrocks with a special edition T-shirt, the MDA enlisted the talent and expertise of freelance graphic designer, Dustin Clark. Dustin, a 28-year-old designer and illustrator living with Becker muscular dystrophy, is passionate about the incredible impact that MDA Shamrocks has had on the neuromuscular community. He was excited to . . .
Researchers at Wave Life Sciences are seeking boys living with DMD caused by gene mutations amenable to exon 53 skipping interventions to participate in a phase 1b/2a clinical trial. This trial will evaluate the safety, effectiveness, and correct dosage of the investigational drug WVE-N531. WVE-N531 is an exon-skipping drug designed to promote skipping over a section . . .
At a time when so much is changing, one thing that remains constant is the Muscular Dystrophy Association’s dedication to providing programs and support to help children and adults with neuromuscular disease live longer and grow stronger. A diagnosis of muscular dystrophy, ALS, or other related neuromuscular disease can bring many challenges to individuals and . . .
As the Muscular Dystrophy Association (MDA) continues our commitment to empowering people living with neuromuscular disease, we are excited to present our 2022 blog series: “Quest for Success”. Success looks different to everyone and this monthly blog will detail the different paths that individuals with neuromuscular disease have taken to reach their potential and the . . .