Researchers at PTC Therapeutics are looking for participants with Duchenne muscular dystrophy to participate in a Phase 3 study. The goal of the study is to evaluate the safety and efficacy of Translarna (ataluren). The drug is believed to promote the formation of full-length and functional dystrophin protein in boys with a nonsense (or premature . . .
Today, MDA, supported by grants from VieMed Healthcare and SYNERGY HomeCare, has released the MDA Guide for Caregivers, a comprehensive resource to empower individuals living with neuromuscular disease and their caregivers. The guide provides practical advice, recommendations, tips and resources to maximize quality of life, drawing on the expertise of numerous MDA Care Center staff. . . .
The Muscular Dystrophy Association recently awarded an MDA human clinical trial grant for the development of a critical biomarker for Charcot-Marie-Tooth disease (CMT) to Mary M. Reilly, professor of clinical neurology and consultant neurologist, UCL Institute of Neurology and National Hospital for Neurology and Neurosurgery, London. The investment, totaling $1 million over three years, will . . .
After significant collaborative work among many stakeholders in the spinal muscular atrophy (SMA) community, we are pleased to share that the Secretary of Health and Human Services (HHS), Dr. Alex Azar, has accepted the recommendation to add SMA to the Recommended Uniform Screening Panel (RUSP) for newborns. This is a landmark decision for the SMA . . .
Former NFL great Dwight Clark died from ALS (amyotrophic lateral sclerosis) on June 4, a little more than one year after revealing he had the disease. Clark was immortalized when he caught the winning touchdown pass in the NFC Championship Game in January 1982, sending the San Francisco 49ers to their first Super Bowl. The . . .
This morning, Summit Therapeutics announced that it is discontinuing development of ezutromid for Duchenne muscular dystrophy after primary and secondary endpoints were missed after 48 weeks of treatment in their PhaseOut DMD trial. PhaseOut DMD was a phase 2, multi-center, open-label clinical trial of ezutromid, a utrophin-modulation therapy. Thirty-eight boys enrolled in the trial completed the 48-week regimen. The . . .
This week, Sarepta Therapeutics announced encouraging preliminary results from a phase 1/2a gene therapy trial designed to assess the investigational drug AAVrh74.MHCK7.micro-Dystrophin in boys with Duchenne muscular dystrophy (DMD). After 90 days in the trial, the first three participants all showed robust expression of micro-dystrophin — a shortened version of the protein that is absent . . .
The Muscular Dystrophy Association recently awarded an MDA clinical research network grant to develop and maintain a core facioscapulohumeral muscular dystrophy (FSHD) Clinical Trial Research Network (CTRN). The investment, totaling $1.2 million over three years, supports seven medical centers that specialize in FSHD research and clinical care and is targeted to spur advances in FSHD . . .
Researchers are looking for people with myasthenia gravis (MG) to participate in a phase 3 clinical trial to evaluate the safety and effectiveness of the investigational drug amifampridine phosphate (brand name Firdapse). Effects of the drug will be assessed in individuals with muscle-specific kinase (MuSK) antibody positive MG and in a smaller group with acetylcholine . . .
Researchers are looking for people with Charcot-Marie-Tooth disease (CMT) to participate in a phase 2 clinical trial to evaluate the safety and effectiveness of the investigational drug FLX-787-ODT. FLX-787-ODT, under development by Flex Pharma, is taken in tablet form and is designed to reduce muscle cramps in adults with CMT. Trial length is approximately three . . .