bluebird bio has produced updated data from its gene therapy for the rare genetic neurological disease cerebral adrenoleukodystrophy (CALD), showing that it continues to work in the long-te
Patients in England and Wales with hereditary angioedema (HAE) should soon have a new treatment option after NICE recommended Takeda’s Takhzyro (lanadelumab) should be funded by the NHS in
Acceleron has axed clinical development of its ACE-083 in the inherited muscle-wasting disease muscular dystrophy, after the drug fell short against efficacy measures in a phase 2 trial.
Orchard Therapeutics has said it plans to file for approval for a gene therapy for the life-threatening rare disease metachromatic leukodystrophy (MLD) in Europe, after publishing new data.
A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.
