gene therapy

News

Orchard’s MLD gene therapy becomes costliest US medicine

Orchard Therapeutics has revealed its US pricing plans for Lenmeldy, its gene therapy for metachromatic leukodystrophy (MLD), placing a $4.25 million price tag on the one-

News

Orchard's gene therapy for MLD finally gets US approval

The FDA has cleared its first gene therapy for kids with metachromatic leukodystrophy (MLD), a rare genetic disorder of the brain and nervous centre.

R&D

Looking to the future of clinical trials: Gene therapy, prec...

Next-generation technologies in medicine have significantly expanded our ability to identify and address rare diseases, propelling forwards the development of more effective and targeted tr

R&D

The opportunities and challenges facing rare disease therapi...

To mark upcoming Rare Disease Day, pharmaphorum asked a panel of experts to give their thoughts on the opportunities and challenges facing developers of rare disease therapies in 2024.

R&D