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 every six months. Each visit . . .
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, . . .
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 . . .
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 . . .
Researchers at Brainstorm Cell Therapeutics are looking for participants with amyotrophic lateral sclerosis (ALS) to participate in a Phase 3 study designed to help researchers evaluate the effects of mesenchymal stromal stem cells secreting neurotrophic factors (MSC-NTF cells) on disease progression in patients with ALS. In ALS, motor neurons degenerate or die, and stop sending . . .
Early identification and treatment for neuromuscular disorders are essential to optimize health outcomes. Newborn screening, which identifies health issues via a blood test taken soon after birth, is essential to ensure that infants born with serious but treatable disorders have the best possible chance at receiving the care and support services they need as early . . .
Researchers at Orphazyme are looking for participants with amyotrophic lateral sclerosis (ALS) to participate in the ORARIALS-01 Phase 3 study. The study is designed to help researchers evaluate the effects of arimoclomol on disease progression in patients with ALS. In ALS, motor neurons degenerate or die and stop sending messages to the muscles, which gradually . . .
Researchers at Catabasis are looking for boys with Duchenne muscular dystrophy (DMD) to participate in a Phase 3 study (PolarisDMD). The goal of the study is to evaluate the safety and efficacy of edasalonexent, which researchers believe may slow down progression of skeletal and cardiac muscle disease in DMD by inhibiting NF-kB. NF-kB is a . . .