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.
R&D SpringWorks spins out of Pfizer on rare disease mission A new biotech spun out of Pfizer is to develop four drugs for rare and serious diseases.
News GBL joins CVC in €10.7bn takeover bid for Recordati A consortium of investors has made a €10.7bn offer to take Italian pharma Recordati private, but some analysts are sceptical the deal will go through.
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