genetic diseases

News

All newborns in England will be screened for rare muscle-wasting disease spinal muscular atrophy from 2027, in a landmark move for patients.

News

Novartis has secured EU approval for Itvisma, a new formulation of its spinal muscular atrophy gene therapy usable in a broader patient population.

News

Novartis' $12bn takeover of Avidity could potentially give it the first therapy for genetic disorder facioscapulohumeral muscular dystrophy.

News

A scheme to hasten the diagnosis of children with genetic disorders is achieving strong results and could be a template for national health systems.

News

Rocket Pharma has won FDA approval for the first gene therapy for LAD-I, a devastating genetic disorder causing severe vulnerability to infections.

R&D

Michelle Werner, Director of Alltrna, discusses her company’s approach of leveraging transfer RNA (tRNA) to shift the paradigm in genetic medicine.