News Biogen bids to extend use of Skyclarys to younger patients Biogen has started a new phase 3 trial that aims to extend the use of its Friedreich's ataxia drug Skyclarys to patients as young as two.
News NHS cleared to use Novo Nordisk's long-acting growth hormone Novo Nordisk's long-acting injectable Sogroya has been backed for NHS use in young people with growth hormone deficiency.
News World first as infant gets personalised CRISPR therapy In a medical breakthrough, a child diagnosed with a rare genetic disorder has received a bespoke CRISPR gene editing therapy to correct the mutation.
News SMA treatment with Evrysdi 'could start prenatally' Should treatment of neurodegenerative disorder SMA with Roche's Evrysdi start even before birth? One case study suggests so.
News Sensorion encouraged by deafness gene therapy data Sensorion's gene therapy for otoferlin-related hearing loss has shown early signs of improving hearing in two toddlers in a phase 1/2 trial.
News MSD eyes June verdict for paediatric RSV antibody The FDA has started a review of MSD's RSV antibody clesrovimab, a potential rival to Beyfortus, with a verdict due in the middle of next year.
Market Access The value of a partner, globally and locally A new podcast looks at recent trends related to drug approvals, as well as challenges and opportunities both small and large pharma companies face