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 Yuliya Matyushenko was 12 years old, the Paris-based rare disease organization Eurordis featured her on a poster promoting Rare Disease Day 2015. In late February, the young woman with spinal muscular atrophy (SMA) was hiding in a bare concrete basement in Kharkiv as Ukraine’s second-largest city, located only 35 kilometers from the Russian border, . . .
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 . . .
Today is the last day of 2022 MDA’s Clinical & Scientific Conference. The theme for today is clinical trials in neuromuscular disease (NMD)! Highlights Best poster winners At the Highlight Session at the end of the day, two awards were presented for outstanding poster presentations. PhD candidate Sarah Holbrook from the Jackson Laboratory at the . . .
Today was the second full day of sessions of 2022 MDA’s Clinical & Scientific Conference. The three major themes of today’s sessions were (1) therapeutic strategies to treat NMDs, (2) clinical trial design and implementation, and (3) optimizing patient care. Highlights Mitochondrial interventions: small molecule and gene therapy Carlos Moraes, PhD, from the University of . . .
Today was the first full day of sessions of MDA’s 2022 Clinical & Scientific Conference. For the first time since the start of the COVID-19 pandemic, clinicians, scientists, policymakers, nonprofit, and industry leaders are convening, primarily in-person, for a dynamic and informative five days in Nashville, TN. With more than 975 in-person and 490 virtual . . .
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 . . .