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On Feb. 14, Sarepta Therapeutics announced that the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) seeking approval under Priority Review for golodirsen (SRP-4053), its drug candidate for treating Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 53. Priority Review status requires the FDA to review the application and . . .
On Mar. 1, the Charcot-Marie-Tooth Association (CMTA) and the Muscular Dystrophy Association (MDA) announced that they have jointly awarded a research grant totaling $276,430 over three years to Kleopas Kleopa, M.D., professor and senior consulting neurologist at the Cyprus Institute of Neurology and Genetics, Cyprus School of Molecular Medicine, in Nicosia, Cyprus. Dr. Kleopa is . . .
Feb. 28 is Rare Disease Day, when the collective rare disease community raises awareness of the conditions with which we live and advocates for access to new novel treatments like Spinraza, the first FDA-approved disease-modifying drug for spinal muscular atrophy (SMA), a rare neuromuscular disease that affects people like 18-year-old Marley Robinson. Spinraza is making . . .
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, . . .
Results of an extension study following an MDA-supported clinical trial showed that surgical removal of the thymus in patients with myasthenia gravis (MG) offered benefits for as long as five years after surgery. The original clinical trial, which was extended for two years, showed patients who had the thymus removed had reduced muscle weakness and . . .
Joining a growing number of companies developing robotic devices for those living with mobility impairments, Ontario’s Trexo Robotics just launched the newest in its line of robotic walking aids specifically for children, the Trexo Home. Unlike the company’s Trexo Plus, a set of wearable robotic legs that attach to a walker for use in clinical . . .
On Feb. 7, Cambridge, Mass.-based Solid Biosciences announced preliminary results from its phase 1/2 clinical trial, called IGNITE DMD, which is designed to assess the safety and efficacy of its lead drug candidate, SGT-001, in children and adolescents with Duchenne muscular dystrophy (DMD). DMD is caused by a mutation in the dystrophin gene on the X . . .
This week, Arkansas Gov. Asa Hutchinson signed into law a bill, HB 1074, that requires the state to add spinal muscular atrophy (SMA) to its newborn screening panel. SMA is the No. 1 genetic cause of death in infants, affecting approximately 1 in 10,000 babies. MDA was proud to advocate for the addition of SMA . . .
Young adults living with Charcot-Marie-Tooth disease: You can help in a new study. Allison Eveler, a senior occupational therapy major at Elizabethtown College in Pennsylvania, is conducting an undergraduate research study to determine the impacts of CMT on the social participation of young adults. Allison, who herself lives with CMT, recognized a need for research . . .
January is a natural goal-setting time. The new year and its fresh calendar to fill out prompt a re-evaluation of how you want to spend your time and your resources. As you’re mapping out your 2019, MDA can fit into your fitness, financial and philanthropic goals — there’s an opportunity for every interest. Volunteer with . . .
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