Neuromuscular diseases

News

A new FDA approval means Novartis now has a gene therapy option for almost everyone with spinal muscular atrophy (SMA), regardless of their age.

News

The ESSENCE trial of Sarepta's Vyondys 53 and Amondys 45 failed to hit its objective, placing their FDA accelerated approvals at risk.

News

Novartis is poised to make one of its largest takeover deals in its history after agreeing to buy RNA specialist Avidity Biosciences for $12bn.

News

It looks like PTC will have to carry out another trial of its drug for Friedreich ataxia before the FDA will consider approval.

News

Novartis has made an offer to acquire RNA therapeutic specialist Avidity Biosciences, according to an unconfirmed report.

News

Roche has revealed good results from a phase 2 trial of its oral BTK drug fenebrutinib in relapsing multiple sclerosis as it waits for phase 3 data.