Yesterday, Biogen released a letter to the ALS community regarding its experimental therapy, tofersen (formerly known as BIIB067), an antisense oligonucleotide being studied for the potential treatment of amyotrophic lateral sclerosis (ALS) in adults with a confirmed superoxide dismutase 1 (SOD1) genetic mutation, which is a subtype of familial ALS that makes up 2 percent . . .
Researchers at PTC Therapeutics are looking for participants with Duchenne muscular dystrophy (DMD) to participate in a Phase 3 study. The goal of the study is to characterize the long-term effects of Translarna (ataluren). The drug is believed to promote the formation of full-length and functional dystrophin protein in boys with a nonsense mutation (which . . .
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 . . .
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 . . .
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 . . .
At MDA, we believe in the power of community and the importance of building relationships with families going through similar experiences. We invite individuals living with a neuromuscular disease, as well as caregivers and loved ones, to attend MDA Engage educational events taking place across the country. MDA Engage Community Education Seminars are one-day events . . .
Researchers at Neurogene are seeking participants living with Charcot-Marie-Tooth disease (CMT) type 4J to participate in a natural history study. This study aims to better understand disease course so researchers will be able to determine clinically meaningful outcome measures for use in future clinical trials. This five-year study will consist of 10 visits (one visit every six months). . . .
Researchers at ReveraGen BioPharma are looking for ambulatory boys with Duchenne muscular dystrophy (DMD) to participate in a Phase 2b study. The goal of the study is to evaluate the safety and efficacy of vamorolone. Researchers hope this therapy may improve muscle strength and endurance with a more favorable safety profile compared to the current . . .
Researchers at Amicus Therapeutics are looking for adults with late-onset Pompe disease (LOPD) to participate in a Phase 3 study. The goal of the study is to evaluate the safety and efficacy of ATB200/AT2221, which researchers hope may improve muscle function and respiratory function compared to the standard of care. Participants will be randomized to . . .
Feb. 13-18, as established by MDA’s colleagues at Parent Project Muscular Dystrophy (PPMD), is Duchenne Muscular Dystrophy Awareness Week, when we recognize both the individuals and families impacted by Duchenne muscular dystrophy (DMD) and the continuing need for innovative treatments — and, one day, a cure — for this disease. In honor of the week, . . .
