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 . . .
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 . . .
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 . . .
Solid Biosciences has announced that the first patient to be treated in its phase 1/2 clinical trial to test SGT-001, the company’s experimental gene therapy treatment for DMD, experienced a serious safety issue that was not among what was predicted based on what is known about the therapy — known as a Suspected Unexpected Serious . . .
Researchers are looking for children and adults with congenital myasthenic syndrome (CMS) to participate in a phase 3 study being conducted by Catalyst Pharmaceuticals to test the experimental drug amifampridine phosphate (brand name Firdapse).
MDA’s 2018 Clinical Conference is the preeminent gathering of MDA Care Center health care providers and other medical and scientific experts from across the country designed to enhance the communication of new medical advances and information relating to the delivery of best-in-class care for individuals with neuromuscular disease.
The Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), a federal committee that oversees newborn screening, voted today to add spinal muscular atrophy (SMA) to the national list of disorders recommended for screening at birth. Following today’s vote, the Secretary of Health and Human Services (HHS) will make a final decision on whether . . .
With the goal to improve diagnosis and care for people living with Duchenne muscular dystrophy (DMD), the Centers for Disease Control (CDC) today published updated Care Considerations for DMD. The comprehensive set of clinical care recommendations, drafted by a steering committee of experts from a wide range of disciplines with support from MDA, offers care providers guidelines for diagnosing DMD and managing its symptoms.
Researchers are looking for individuals with SMA type 2 to participate in the phase 1 STRONG clinical trial, to test safety, dosing and proof of concept for efficacy for its SMA gene replacement therapy AVXS-101.