As scientists explore treating genetic disorders before birth, the promise of foetal gene therapy is growing—but there are ...

Public health activists estimate Roche earned significantly more from risdiplam, a spinal muscular atrophy drug, than its development costs. Roche disputes this, citing expenses from unsuccessful ...

While Roche’s oral risdiplam has been tipped by some analysts to be a big contender in the market for spinal muscular atrophy (SMA) treatments, Novartis’ research chief Jay Bradner has a ...

The FDA has started a priority review of Roche’s spinal muscular atrophy (SMA) candidate risdiplam, with a decision due by 24 May next year. If approved, risdiplam will be the third drug to be ...

He had a hunch that the oral SMA treatment risdiplam, made by Roche, had prenatal potential because it’s safe for kids and ...

0.51 points in the sham control arm. In addition, in the Phase IIIb STRENGTH study, treatment with OAV101 IT in patients who have discontinued treatment with nusinersen or risdiplam demonstrated ...

In February 2025, researchers at St Jude Children’s Research Hospital reported the first known in utero use of Roche’s spinal muscular atrophy (SMA) drug Evrysdi (risdiplam) in a human foetus.

The drug in question is risdiplam, a medication for spinal muscular atrophy (SMA). The report on Roche's profit estimates are based on evaluations by public health advocates. Following Roche's ...

PTC Therapeutics, Inc. today announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington's disease (HD) patients. The study met its primary endpoint of ...