Gene therapy medication, Spinraza, provides improvements for patients with spinal muscular atrophy

Spinraza has provided significant improvements in cases with the next most severe form of the neuromuscular disease, spinal muscular atrophy (SMA), which affects children aged from 6 to 18 months.   “The effects are convincing, and this reinforces results from the earlier study,” says Mar Tulinius, Professor of paediatrics at Sahlgrenska Academy and chief medical

Continue Reading