Since the early 1950s, not long after the Muscular Dystrophy Association’s formation, America’s young people living with muscular dystrophy and related neuromuscular diseases have stepped forward to share their stories, raise awareness of the need for treatments and cures for rare diseases, and represent MDA’s mission with humanity and grace. More than 40 MDA National . . .
MDA’s first-ever combined Clinical & Scientific Conference kicked off today at the Hyatt Regency Orlando in Florida. MDA President & CEO Lynn O’Connor Vos and MDA Chairman of the Board Dr. R. Rodney Howell opened the event. “MDA is proud to convene this esteemed group, and we are grateful that you have joined us for . . .
The Muscular Dystrophy Association (MDA) is pleased to announce a collaboration with the Broad Institute of MIT and Harvard, which aims to bring genetic diagnoses to more individuals living with limb-girdle muscle weakness. The goal of the LGMD Rare Genomes Project is to bring the most advanced genetic techniques, including whole-genome sequencing and genetic variation . . .
With a laser focus on new horizons in neuromuscular research and care, the Muscular Dystrophy Association (MDA) is combining its clinical and scientific conferences for the first time, establishing a robust, state-of-the-art gathering for both clinicians and investigators from across the nation. Medical and scientific experts in neuromuscular disease from academia, private practice, government, and . . .
On April 8, Audentes Therapeutics announced that it will expand its gene-targeted therapy platform to include developing experimental drugs for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). The company has entered into a licensing agreement with Nationwide Children’s Hospital in Ohio and will collaborate with leading researchers in the field, Kevin M. Flanigan, . . .
To kick off 2019 National Volunteer Month, the Muscular Dystrophy Association was honored to welcome Marriott International volunteers from all over the world into our Chicago offices on Friday, April 5, as part of Marriott’s Serve 360 event. Continuing its tradition of making its communities better places to live, work, and visit, Marriott International this . . .
Twin brother and sister Michael and Amy Schleicher’s story starts where a lot of kids’ stories do. They looked up to their big brother, Matt, and when he did something cool, Michael and Amy wanted to do it, too. In the case of the fateful summer that would point the twins in a long-term direction, . . .
Today, the Muscular Dystrophy Association (MDA) announced the awarding of eight new grants totaling more than $2 million toward research focused on amyotrophic lateral sclerosis (ALS), a disease in which muscles become weak and eventually nonfunctional. Since its inception, MDA has invested more than $165 million in ALS research, and in the last five years . . .
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 . . .
On Mar. 28, Sarepta Therapeutics announced encouraging interim results from a phase 3 clinical trial that suggest the experimental drug casimersen potentially may be effective as a treatment for Duchenne muscular dystrophy (DMD). Earlier this year, the U.S. Food and Drug Administration (FDA) accepted Sarepta Therapeutics’ New Drug Application (NDA) seeking approval under Priority Review for . . .