Novartis gene therapy helps children with rare muscle disorder in study

Swiss drugmaker Novartis said on Monday its gene therapy helped improve motor function in children with a rare muscle disorder that leaves patients too weak to walk, talk and swallow.

Novartis was testing the therapy, onasemnogene abeparvovec or OAV101 IT, in a late-stage trial of patients between the ages of 2 and less than 18 with spinal muscular atrophy (SMA).

The condition prevents the body from producing a protein necessary for neuromuscular development. The Swiss drugmaker's one-time therapy, branded as Zolgensma, is already approved in the US to treat children less than 2 years of age with the condition.