Spinal muscular atrophy (SMA) is a rare hereditary neuromuscular disorder in which the spinal cord does not form normally.

Because sickle cell disease isn’t as progressive a disease as others that have gene therapies available, there isn’t as much ...

The babies were treated before symptom onset, and are sitting, standing, and walking independently, many consistent with ...

Two years after treatment, both girls show normal motor function and no signs of spinal muscular atrophy symptoms.

Majority of children with SMA treated with Roche’s Evrysdi are able to sit, stand and walk independently, two-year data demonstrate ...

Analyst Jason Gerberry of Bank of America Securities maintained a Buy rating on Biohaven Ltd. (BHVN – Research Report), retaining the ...

The findings are significant for the liver research community and others interested in the gut-liver axis. (A) AOAH cleaves the two piggyback fatty acyl chains from the lipid A moiety, inactivating ...

Roche has announced positive two-year data from its RAINBOWFISH study, showcasing the effectiveness of Evrysdi® (risdiplam) ...

Scientists compared the CASMA screening approach against standard methods, and found it better at detecting problems in genes ...

Biohaven has surged driven by promising data from its troriluzole trials for spinocerebellar ataxia and ongoing OCD studies.