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 . . .
Researchers are looking for people with myasthenia gravis (MG) to participate in a multinational phase 2A clinical trial to evaluate the safety, tolerability and effectiveness of the investigational drug UCB7665. UCB7665, under development by UCB Pharma, is administered via subcutaneous (under-the-skin) injection and is designed to reduce the severity of muscle weakness and fatigue in . . .
With a focus on respiratory care in Duchenne muscular dystrophy (DMD), Santhera Pharmaceuticals has made available new resources for individuals with the disease. In addition, the company is seeking individuals to participate in a phase 3 clinical trial to test a respiratory drug in people with DMD. SIDEROS trial seeks participants Researchers are looking for . . .
MDA has awarded a human clinical trial grant totaling $750,000 to ALS ONE to explore the potential for a type of imaging called positron emission tomography (PET) to measure inflammation in the brain that could serve as a biomarker for ALS. ALS ONE is an alliance between four institutional leaders in ALS treatment development: Massachusetts . . .
MDA has announced the award of 13 new grants totaling $2.6 million as part of its continued commitment to finding treatments and cures for the diseases in its program. Each of the 13 new grants funds a project that aims to increase our understanding of the causes or underlying mechanisms of neuromuscular disease or potential . . .
A phase 1 trial to test omigapil in congenital muscular dystrophy (CMD) conducted at the National Institutes of Health is complete and data indicate it met its primary objective. Results from the trial, also known as CALLISTO, show a favorable pharmacokinetic profile of omigapil and demonstrated that the drug was safe and well-tolerated in children and . . .
Catalyst Pharmaceuticals has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the investigational drug amifampridine phosphate (brand name Firdapse) for the treatment of people with Lambert-Eaton myasthenic syndrome (LEMS). The FDA has 60 days to determine whether the NDA is complete and acceptable for filing. If the FDA . . .
The Tuesday afternoon sessions of MDA’s 2018 Clinical Conference focused on best practices in newborn screening. Augmentative communication, pediatric-to-adult transition, respiratory health and care coordination also were covered in parallel sessions. Among the highlights: Newborn screening R. Rodney Howell, M.D., from the Miller School of Medicine, University of Miami, talked about the history of newborn . . .
