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, . . .
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 . . .
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 . . .