Today, the U.S. Food and Drug Administration (FDA) approved Zolgensma (onasemnogene abeparvovac-xioi), the first gene therapy for a neuromuscular disease. Zolgensma is a one-time intravenous (into the vein) infusion for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene, . . .
Earlier this week, JAMA Neurology published an article co-authored by Muscular Dystrophy Association (MDA) staff members that addresses the role of newborn screening (NBS) in the field of neuromuscular disease. In the article, “Maximizing the Benefit of Life-Saving Treatments for Pompe Disease, Spinal Muscular Atrophy, and Duchenne Muscular Dystrophy Through Newborn Screening: Essential Steps,” the . . .
Dr. Jonathan Glass is the director of the Emory ALS Center, co-chair of the Northeast ALS Consortium, and professor of Neurology and Pathology at Emory University in Atlanta. Dr. Glass’ laboratory focuses on the study of amyotrophic lateral sclerosis (ALS), investigating causes and potential therapies and cures. MDA has been a partner in research, providing . . .
It’s a hard fact that only three medications have been approved to treat amyotrophic lateral sclerosis (ALS) in nearly 25 years. Fortunately, research in ALS has exploded in the past decade, and new technologies have enabled the development of gene-targeting therapies such as gene replacement, gene silencing, and gene editing. MDA has always been committed . . .
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 . . .
We are pleased to announce that on May 1, 2019, the American Society for Gene and Cell Therapy awarded MDA the Sonia Skarlatos Public Service Award for 2019. Named for its inaugural co-recipient and tireless gene therapy advocate, the ASGCT’s Sonia Skarlatos Public Service Award recognizes a person or group that has consistently fostered and . . .
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 . . .
MDA’s first-ever combined Clinical & Scientific Conference kicked off today at the Hyatt Regency Orlando in Florida. MDA President & CEO Lynn O’Connor Vos and MDA Chairman of the Board Dr. R. Rodney Howell opened the event. “MDA is proud to convene this esteemed group, and we are grateful that you have joined us for . . .
