Nicole Wetsman,  —

RG7916 Safe for Infants With Type 1 SMA, Early Data Indicate

Early interim data from a Phase 2 clinical trial evaluating varying doses of a potential oral therapy, RG7916, in infants with type 1 spinal muscular atrophy (SMA) is showing good safety and tolerability, researchers said in a recent scientific presentation. Preliminary findings of 13 babies enrolled…

Infant Motor Function Improves With Longer Spinraza Treatment, New Analysis Reports

Infants with spinal muscular atrophy (SMA) continue to show better motor control and strength the longer they receive Spinraza (nusinersen), concludes a new and long-term analysis of final data from a Phase 3 clinical trial in babies with type 1 disease. These infants are also less likely to be…