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
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
Cancer Research UK (CRUK) has said it will award £123 million ($150 million) in funding over the next seven years to Scotland’s Beatson Institute, which will be renamed th
To improve trial recruitment, engagement, and retention, and to increase the likelihood that trials reflect what matters most to patients, regulatory authorities increasingly point to metho