NICE has recommended that PTC Therapeutics’ gene therapy for ultra-rare disease aromatic L-amino acid decarboxylase (AADC) deficiency Upstaza be made available for NHS use
Bristol-Myers Squibb’s Opdivo has become the first immunotherapy to be cleared for NHS use alongside chemotherapy as a pre-surgery (neoadjuvant) therapy for resectable non
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.
Chinese biotech Hutchmed has completed its rolling marketing application in the US for oral VEGF receptor inhibitor fruquintinib, which was licensed by Takeda for a hefty