News uniQure presses 'refresh' on haemophilia gene therapy, gets ... Biotech has been in talks with US and EU regulators
Views & Analysis Rare disease market research needs a multifaceted approach Challenges in rare disease market research and the role of online research.
News FDA experts to vote on Spark's groundbreaking gene therapy Luxturna could be single life-changing injection
News Ablynx soars after blood clot trial success European regulators are already reviewing caplacizumab
News PTC Therapeutics fails to convince FDA panel about DMD drug FDA advisers have voted against approval for PTC Therapeutics’ Duchenne drug Translarna because of doubts about its efficacy.
News FDA reviewers slam PTC's Duchenne drug ahead of meeting FDA staff not persuaded by patchy clinical trial evidence for Translarna.
News Ultragenyx to slash costs after Angelman trial failure Ultragenyx's therapy for the rare genetic disorder Angelman syndrome, apazunersen, has flunked a late-stage trial, crashing its share price.
Digital Sponsored Discover a new era of healthcare panel engagement: Grounded ... A new webinar from Konovo will address some of the critical topics impacting today’s healthcare market research industry.
