Trial to Test MG Drug UCB7655 Seeks Participants

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 . . .

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Santhera Seeks Participants for DMD Study, Makes Available EAP and Online DMD Resource

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 . . .

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MDA and ALS ONE Work to Advance ALS Biomarker Research

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 . . .

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13 New MDA Grants Totaling $2.6 Million Push for More Progress

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 . . .

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Encouraging Results in Phase 1 CALLISTO Trial to Test Omigapil in CMD

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 . . .

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Catalyst Pharmaceuticals Submits New Drug Application to FDA for Firdapse to Treat LEMS

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 . . .

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2018 MDA Clinical Conference Highlight Report: Day 2 Afternoon Sessions

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 . . .

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2018 MDA Clinical Conference Highlight Report: Day 2 Morning Sessions

On Tuesday morning, MDA’s 2018 Clinical Conference sessions focused on best practices in cardiac care, physical therapy, bone health, technology and nutrition. Among the highlights: Cardiac management for the muscular dystrophy lifespan Elizabeth McNally, M.D., Ph.D., from Northwestern University Feinberg School of Medicine, noted that to best manage the heart in neuromuscular disease, a genetic . . .

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2018 MDA Clinical Conference Highlight Report: Day 1 Afternoon Sessions

Monday’s afternoon sessions at MDA’s 2018 Clinical Conference focused on advances in personalized medicine, including the latest in precision therapies, genomics, digital health and MDA’s MOVR initiative. Among the highlights: Correction of Duchenne muscular dystrophy by genome editing Leonela Amoasii, Ph.D., from UT Southwestern Medical Center, discussed a vision for gene editing in DMD. The . . .

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2018 MDA Clinical Conference Highlight Report: Day 1 Morning Sessions

Monday’s morning sessions at MDA’s 2018 Clinical Conference focused on what’s new in ALS (amyotrophic lateral sclerosis), Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD), myotonic dystrophy (DM) and myasthenia gravis (MG). Among the highlights: ALS  James D. Berry, M.D., M.P.H., from Massachusetts General Hospital, noted that, “The really profound answer to what’s new in . . .

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