Three men with late-onset spinal muscular atrophy (SMA) on Evrysdi treatment were able to conceive with their partners, per a ...

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 ...

Anna Landre, a college student who lives with spinal muscular atrophy, found herself fighting another battle when her insurer informed her they were reducing the number of hours they would cover ...

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 ...

for apitegromab for the improvement of motor function in patients with spinal muscular atrophy (SMA). Apitegromab, a fully human monoclonal antibody, works by selectively binding to the pro- and ...

Results from the STEER and STRENGTH studies showed that OAV101 IT led to a 2.39-point improvement in motor function in patients with spinal muscular ... type I, there is a 95% mortality rate by 18 ...

Apitegromab is an investigational treatment aimed at improving motor function for people living with spinal muscular atrophy. Scholar Rock said it has also submitted and received validation for its ...