Hosted on MSN2d
Broader population could benefit from Novartis spinal muscular atrophy treatment
Positive results from its Phase III STEER study mean Novartis could boost sales of its OAV101 IT drug for spinal muscular ...
PMLiVE13d
Novartis’ investigational spinal muscular atrophy gene therapy shows promise in late-stage study
Novartis has shared positive top-line results from a late-stage study of its investigational gene therapy in patients aged two to less than 18 years with the rare neuromuscular disease spinal muscular ...
Hosted on MSN16d
Novartis’ Phase III spinal muscular atrophy therapy trial meets primary endpoint
Novartis has reported positive topline outcomes from the Phase III STEER trial of an investigational gene therapy, ...
Nature7y
Medical Ethics: Genetic Testing and Spinal Muscular Atrophy
The mutation that causes Spinal Muscular Atrophy (SMA) can be detected in a prenatal screen. But a parent's decision to test for this terrible disease is not as easy as you would think.