On May 6, the U.S. Food and Drug Administration (FDA) announced the approval of Ruzurgi (amifampridine) for the treatment of children with Lambert-Eaton myasthenic syndrome (LEMS) who are between 6 and 17 years of age. This is the second FDA-approved treatment for LEMS and the first approved treatment for pediatric LEMS patients. Ruzurgi is an oral potassium . . .
In April, MDA held its first ever combined Clinical & Scientific Conference, themed “Progress in Motion,” in Orlando Fla. Clinicians, scientists, policymakers, nonprofit, and industry leaders convened for a dynamic and informative five days in Orlando, Fla. With more than 1,200 attendees, 23 sessions, 136 presentations, and 300 posters, this was MDA’s largest conference ever. . . .
Yesterday, Biogen released a letter to the ALS community regarding its experimental therapy, tofersen (formerly known as BIIB067), an antisense oligonucleotide being studied for the potential treatment of amyotrophic lateral sclerosis (ALS) in adults with a confirmed superoxide dismutase 1 (SOD1) genetic mutation, which is a subtype of familial ALS that makes up 2 percent . . .
In a study conducted in mice, scientists led by an MDA-funded researcher found that increasing the amount of a certain mitochondrial membrane protein, mitofusin-1 (MFN1), lessened symptoms of Charcot-Marie-Tooth disease (CMT) type 2A. The findings suggest that it may be possible to treat CMT2A using gene-replacement therapies that deliver functional copies of missing or mutated . . .
The Muscular Dystrophy Association (MDA) is pleased to announce a collaboration with the Broad Institute of MIT and Harvard, which aims to bring genetic diagnoses to more individuals living with limb-girdle muscle weakness. The goal of the LGMD Rare Genomes Project is to bring the most advanced genetic techniques, including whole-genome sequencing and genetic variation . . .
With a laser focus on new horizons in neuromuscular research and care, the Muscular Dystrophy Association (MDA) is combining its clinical and scientific conferences for the first time, establishing a robust, state-of-the-art gathering for both clinicians and investigators from across the nation. Medical and scientific experts in neuromuscular disease from academia, private practice, government, and . . .
Today, the Muscular Dystrophy Association (MDA) announced the awarding of eight new grants totaling more than $2 million toward research focused on amyotrophic lateral sclerosis (ALS), a disease in which muscles become weak and eventually nonfunctional. Since its inception, MDA has invested more than $165 million in ALS research, and in the last five years . . .
On Mar. 5, the U.S. Food and Drug Administration (FDA) awarded Orphan Drug designation to Acceleron Pharma’s ACE-083, a locally acting muscle agent, for treating Charcot-Marie-Tooth disease (CMT). Delivered by intramuscular injection, ACE-083 is based on the naturally occurring protein follistatin and is designed to enhance the body’s own promoters of muscle growth, specifically in . . .
Young adults living with Charcot-Marie-Tooth disease: You can help in a new study. Allison Eveler, a senior occupational therapy major at Elizabethtown College in Pennsylvania, is conducting an undergraduate research study to determine the impacts of CMT on the social participation of young adults. Allison, who herself lives with CMT, recognized a need for research . . .
