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, . . .
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 . . .
Did you know that according to a recent MDA survey, only 21% of adults living with neuromuscular disease are working part or full-time? Many individuals with disabilities have a desire to work but face employment hurdles, including the lack of wraparound support services, inaccessible workplaces, discriminatory wages, and a potential loss of Supplemental Security Income . . .
The individuals and families in our neuromuscular community have always been the heart of MDA’s mission. MDA’s Family Support Team executes a variety of programs that empower individuals and families on their journeys. As another year ends, and the new year is ramping up, the Family Support Team is proud to reflect on services provided . . .
MDA’s Ambassadors are pivotal to our mission: empowering people living with muscular dystrophy, ALS, and related neuromuscular diseases to achieve their potential. We do that through care, research, and advocacy. Because the mission is big, we need big partners who can help evangelize what MDA is, what we do, and, most importantly, who we do it for. Enter, . . .
It’s a new year and MDA’s commitment to transforming the lives of people living with neuromuscular disease through advocacy remains a top priority. We accomplished a lot last year, but we still have many vital goals to accomplish in 2022. We are excited to share a sneak peek at the advocacy team’s agenda for the . . .
For more than 70 years, the Muscular Dystrophy Association (MDA) has been committed to empowering people living with muscular dystrophy, ALS, and related neuromuscular diseases to reach their potential. Through the decades, MDA has led the way through our innovations in care, research, and advocacy. This year, we go bigger. In an interview with . . .
On Dec. 17, the US Food and Drug Administration (FDA) granted approval to efgartigimod (Vyvgart) for the treatment of generalized myasthenia gravis (gMG) in adults who test positive for the anti-acetylcholine receptor (AChR) antibody. To date, Vyvgart is the second disease modifying drug approved to treat gMG. Vyvgart will be made available in the United States . . .
On Oct. 28, 2021 the Muscular Dystrophy Association (MDA) announced the awarding of 18 new grants totaling over $1.6 million toward neuromuscular disease (NMD) research. These new grants represent a continued commitment by MDA to fund groundbreaking research that will one day lead to treatments and cures for the diseases in its program. The newly . . .