Views & Analysis LHON in the family: living under the spectre of an hereditar... It’s hard to imagine suddenly losing your sight in your 20s, but that’s what happened to Richard Wheeler when he developed
Views & Analysis Out of the darkness: new gene therapies for eye diseases US FDA expert committee’s ‘yes’ could pave the way for a new generation of gene therapies for rare inherited eye disorders.
Views & Analysis Getting a rarer perspective Benefitting from insights from focus groups with rare disease patients.
News Gene therapy firm Kate Tx snapped up by Novartis for $1.1bn Novartis buys gene therapy specialist Kate Therapeutics for $1.1 billion, adding an early-stage rival to Sarepta's muscular dystrophy therapy Elevidys
Sales & Marketing Coming soon: The Life Sciences Industry Report 2025 – insigh... Prepare for 2025 with pharmaphorum’s Life Sciences Report, featuring expert insights on AI in drug development, gene therapy, and top industry trends