News Mixed data mars Sanofi's venglustat programme Sanofi has reported positive phase 3 results with venglustat in Gauder 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 NHS "isn't delivering equitable care for rare diseases" A Genetic Alliance UK report claims the NHS is failing to meet the needs of many of the 3.5 million people in the country who live with rare diseases.
Sales & Marketing Sponsored Using AI-driven synthetic personas to take your insights fur... As AI continues to reshape the healthcare landscape, pharma teams are beginning to leverage synthetic personas.
