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.
A mid-stage trial of 4D Molecular Therapeutics gene therapy for eye disease age-related macular degeneration (AMD) 4D-150 has shown promising signs of efficacy, plus a red
Eli Lilly’s acquisition of gene therapy specialist Akouos seems to have paid off, after the lead candidate in the deal restored hearing in a child with congenital deafness