Investigators say thrombospondin-4 levels were reduced in patients with symptoms of spinal muscular atrophy (SMA), but levels ...

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

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 ...

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

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

King’s College London, Guy’s and St Thomas’ NHS Foundation Trust and King’s College Hospital NHS Foundation Trust have been ...

Amwins Group Benefits is now expanding its Gene Therapy Solutions (GTS) program to 15 gene and cell therapies, up from the ...

King's College London, Guy's and St Thomas' NHS Foundation Trust and King's College Hospital NHS Foundation Trust have been ...

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

This week will see the return of the Assisted Dying Bill to Parliament, as it is formally introduced to the House of Commons.