Johnson & Johnson's nipocalimab could become the first approved treatment for a blood disorder that can lead to the death of a foetus or newborn, if positive results i
4D Molecular Therapeutics (4DMT) has said a trial of its gene therapy for Fabry disease has been placed on a clinical hold by the FDA, a few weeks after it reported three
Ipsen has suffered a setback in its marathon effort to bring palovarotene to market for ultra rare disease fibrodysplasia ossificans progressiva (FOP), after EU advisors recommended against
Alnylam's gene-silencing drug Amvuttra has been recommended as a treatment for hereditary transthyretin-related (ATTR) amyloidosis by NICE, paving the way for the drug to be made available
