PTC Therapeutics

PTC Therapeutics has shared interim data from the 12-week portion of the PIVOT-HD Phase II study of PTC518 in Huntington’s disease (HD) patients. The study demonstrated dose-dependent lowering of Huntingtin (HTT) protein levels in peripheral blood cells, reaching a mean 30% reduction in mutant HTT levels at the 10mg dose level. In addition, PTC518 exposure […]

The National Institute for Health and Care Excellence (NICE) has published its Final Guidance recommending Translarna (ataluren) for reimbursement and use across the National Health Service (NHS) in England and Wales.   Translarna is the only approved treatment for patients with nonsense mutation Duchenne muscular dystrophy aged two years and older who can walk.  The final […]