Partner Content Partner Content Official Agenda released for 8th annual Orphan Drugs and Rar... Agenda released for 8th Orphan Drugs and Rare Diseases
News US lawmakers back 'right to try' legislation US lawmakers have approved new laws giving terminally ill patients the “right to try” unapproved drugs if there are no alternatives.
News Spark/Pfizer eye phase 3 trial of haemophilia B gene therapy Spark Therapeutics has already revolutionised treatment of a rare hereditary eye disease with a gene therapy, but could it be about to do the same with haemophilia B?
News Roche haemophilia data piles pressure on Shire Hemlibra threatens to take market share from Advate
Partner Content Partner Content OPEN Health invest in rare disease OPEN Health invest in rare disease
News Solomon quits as Akari CEO following credit card probe Biotech veteran quits after less than a year in charge
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.
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