Roche has highlighted new data with its oral treatment for spinal muscular atrophy (SMA) – Evrysdi – showing its benefits when given to pre-symptomatic babies with the rare disease.
The FDA has relaxed a clinical hold on clinical trials of Novartis' intrathecal formulation of spinal muscular atrophy (SMA) gene therapy Zolgensma, allowing a new phase 3 trial to get unde
NICE has rejected routine NHS funding for Roche's Evrysdi (risdiplam) for spinal muscular atrophy drug in draft guidance, a decision which doesn't come as a surprise to patient association
Novartis has said its gene therapy pricing model, involving a steep one-off payment, could be a template for the hundreds of similar treatments that could follow its Zolgensma for Spinal Mu
After more than eight years at the helm of US biotech Scholar Rock, Nagesh Mahanthappa is standing down as CEO, to be replaced by biopharma industry veteran Tony Kingsley.<
GSK's severe asthma therapy Nucala could find a new lease of life as a treatment for chronic obstructive pulmonary disease (COPD) after showing a clear benefit in a phase 3 trial.<