TerSera Therapeutics LLC today announced that it has completed the previously announced sale of its Infusion Specialty Therapies Business Unit (IST), including two specialty medications, Prialt(R) ...

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 underway.

Bönnemann, who specializes in pediatric neuromuscular and neurogenetic disorders, comes to St. Jude from the National Institutes of Health, where he served as a physician-researc ...

Insmed has launched a first-in-human Phase 1 trial testing its experimental ALS gene therapy INS1202 in people with the disease.

New ASH guidelines outline optimal management for a subgroup of patients with leukemia that historically has been challenging to treat.Two papers published simultaneously in Blood Advances address ...

Novartis has a much-anticipated approval for its spinal muscular atrophy (SMA) treatment onasemnogene abeparvovec in the US, getting a green light for a new version that can make more patients ...

Eliana Merle Barclays Bank PLC, Research Division. Great. Good afternoon, everyone. I'm Ellie Merle, one of the biotech analysts here at Barclays. Very happy to have Ionis here wi ...

Results show that targeting the underlying cause of Dravet syndrome with zorevunersen may improve outcomes for people with this rare, devastating genetic neurodevelopmental dis ...

Sjögren-Larsson syndrome (SLS) constitutes a rare genetic disorder manifesting as a complex neurocutaneous condition characterised by congenital ichthyosis, progressive neurological impairment and ...

Salanersen, a treatment candidate for spinal muscular atrophy (SMA) now in clinical testing, is safe and was shown to stabilize or improve motor function over at least one year of follow-up in ...

The company's clinical development plan comprises three global Phase III studies, including two focused on infants and a third for older patients.

Biogen has launched a trio of Phase 3 trials to test salanersen, a new SMA treatment that aims to be a more convenient ...