News FDA clears Denali's 'game-changer' Hunter syndrome drug Denali's Avlayah is the first drug for neurological complications associated Hunter syndrome to be approved in the US.
News Mixed data mars Sanofi's venglustat programme Sanofi has reported positive phase 3 results with venglustat in Gaucher disease, but the drug missed the mark in a Fabry disease trial.
News Azafaros bags €132m for rare neuro-metabolic disorder drugs Azafaros is set to start phase 3 trials of its lead drug for a trio of rare lysosomal storage disorders, backed by €132m in new Series B financing.
Digital Sponsored Digital health technologies and rare diseases: Enhancing the... This pre-recorded webinar was fully funded by Chiesi GRD and hosted by pharmaphorum. Professor Maurizio Scarpa received an honorarium for his participation and contribution to this webinar.
Digital Sponsored Enabling Digitally Enhanced Care for Rare Diseases in Europe Integrating AI and Connected Care Technologies in Lysosomal Storage Disease Patient Journeys
News OpenBind unveils its first AI model for drug discovery A research consortium that aims to make the UK a leader in AI-driven drug discovery, OpenBind, has hit its first major milestone.
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.
