A higher dose of Spinraza may be more effective than the currently approved dosing schedule for maintaining motor function in ...

Novartis announces positive data from phase III study of intrathecal onasemnogene abeparvovec in broad patient population with SMA: Basel Friday, March 21, 2025, 11:00 Hrs [IST] N ...

Novartis (NVS) announced positive safety and efficacy results from the Phase III program for investigational intrathecal onasemnogene ...

Incorporating systems thinking into drug value assessment promises more realistic evaluations of the impact of new therapies, ...

Disease-modifying therapies (DMTs) may help lower the risk of children’s bone fractures among young patients with spinal muscular atrophy (SMA), a study by U.S. researchers suggests. “Drug therapy led ...