News Can Spark make a profit on its groundbreaking gene therapy? Spark Therapeutics is on the cusp of gaining the first ever approval for a gene therapy from the FDA, a groundbreaking treatment which restores vision to children and adults with a rare inh
News Breakthrough status for BioMarin ratchets up haemophilia A c... The FDA has granted Breakthrough Therapy Designation to BioMarin’s gene therapy for haemophilia A, helping to intensify competition in the field.
Views & Analysis Out of the darkness: new gene therapies for eye diseases US FDA expert committee’s ‘yes’ could pave the way for a new generation of gene therapies for rare inherited eye disorders.
Views & Analysis Addressing market access obstacles for rare bleeding disorde... Scrutiniseing the market access situation for people with rare bleeding disorders.
News uniQure presses 'refresh' on haemophilia gene therapy, gets ... Biotech has been in talks with US and EU regulators
News Spark's groundbreaking gene therapy on cusp of US approval Luxturna wins unanimous backing from FDA advisers
News UK offers meningitis jab to young people after outbreak The UK government has responded to calls to broaden vaccinations for meningitis B, offering the jab to "thousands" of young people this summer.
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.