Novartis is expected to present the latest results in October from its ongoing Str1ve study, which is monitoring the effects of its gene therapy AVXS-101 on 15 infants with spinal muscular atrophy. The company has also been working closely with researchers at Massachusetts General Hospital and Boston Children’s Hospital, where it will be conducting a clinical trial for children who have an inherited form of Duchenne Muscular Dystro@@
Novartis is expected to present the latest results in October from its ongoing Str1ve study, which is monitoring the effects of its gene therapy AVXS-101 on 15 infants with spinal muscular atrophy. Rivals Biogen and Roche will be anxious to know if Novartis has built on its last set of impressive results in April, which saw six infants' motor function increase by an average of 7.8 points on the so-called Chop-Intend scale, one month after treatment, while no "events" – deaths or a need for near-permanent breathing support – were recorded.