Despite significant advancements in spinal muscular atrophy (SMA) management, unmet needs persist, especially for patients ...

Amino acid balance could play a role in SMA, as a study showed acids able to influence nervous system function were ...

The FDA has accepted for Priority Review the BLA for apitegromab for the improvement of motor function in patients with spinal muscular atrophy.

Novartis has announced positive results from a late-stage programme of its experimental gene replacement therapy in patients with the rare neuromuscular disease spinal muscular atrophy (SMA). The ...

this report provides important new evidence for linkage of an X-linked infantile spinal muscular atrophy locus to Xp11.3-Xq11.1. Unlike classical SMA congenital contractures have been associated ...

Nearly six years ago, the Food and Drug Administration approved Zolgensma, a Novartis gene therapy for the fatal neuron-wasting disease spinal muscular atrophy. It heralded a new era of genetic ...