News Sarepta slumps on Elevidys patient death The death of a patient treated with Sarepta's Duchenne muscular dystrophy gene therapy Elevidys has hit its share price.
R&D On incentivisation and investment in rare disease R&D To mark Rare Disease Day, pharmaphorum spoke with Paola Pozzi, partner of telethon strategy at Sofinnova Partners, about the outlook for investment in this field of research.
News Spain agrees to reimburse Orchard's gene therapy Libmeldy Spain has agreed to reimburse Orchard Therapeutics' ex vivo gene therapy for the rare childhood disease metachromatic leukodystrophy.
News Evidence builds behind Regeneron's deafness gene therapy Ten out of 11 children with congenital deafness treated with a gene therapy developed by Regeneron have seen "notable" improvements in hearing.
News Pfizer drops Beqvez, leaving its gene therapy cupboard bare Pfizer has abandoned its haemophilia B treatment Beqvez in all world markets, saying weak demand made the business non-viable.
News Remepy's hybrid Parkinson's therapy poised for phase 3 Remepy's Hybridopa therapy for Parkinson's, which combines an app with levodopa therapy, has shown efficacy in a phase 2 pilot study.
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