Jacifusen was generally well tolerated and reversed disease progression in some people with ALS caused by mutations in the ...

An algorithm is helping to reduce the number of unnecessary hospital admissions for children with sickle cell.

CAR T-cell therapy, a type of treatment that weaponizes the body’s immune system and was originally developed to treat cancer ...

Pepgen will stop development of its Duchenne MD exon-skipping therapy PGN-ED051 after increases in dystrophin levels proved ...