Recent data reveals a concerning trend as UK drops from 2nd to 10th place in European rankings for orphan medicine availability, signaling declining access to rare disease treatments.
PTC Therapeutics' Upstaza has secured CHMP recommendation as the first disease-modifying treatment for AADC deficiency, marking a milestone as the first gene therapy administered directly to the brain.
Drug Development Updates
Stay informed with timely notifications on clinical trials and research advancements.