Roche and PTC Therapeutics' Evrysdi for spinal muscular atrophy (SMA) has been approved by the FDA for younger children with the rare disease, extending its use to include infants less than
PTC Therapeutic's Upstaza gene therapy for patients with genetic disorder AADC deficiency has been recommended for approval in the EU, setting up another test of the commercial prospects f
A disparity in UK access to a medicine for Duchenne muscular dystrophy (DMD) has been addressed, after Scotland gave a green light to use of PTC Therapeutics' Translarna for the first time.
US biotech Sarepta's shares jumped by as much as 60% after a gene therapy produced dramatic results in three boys with the muscle wasting disease Duchenne muscular dystrophy (DMD).
