Gene therapy medication provides improvements in severe SMA – European Pharmaceutical Review
European Pharmaceutical Review |
Gene therapy medication provides improvements in severe SMA
European Pharmaceutical Review Spinraza, the gene therapy medication, also provides significant improvements in cases with the next most severe form of the neuromuscular disease, spinal muscular atrophy (SMA), which afflicts children from 6 to 18 months of age. “The effects are … |
