News PTC’s gene therapy for rare disease cleared for NHS use Upstaza has shown impressive clinical results in children with fatal disorder AADC deficiency.
Digital AI technology: A game-changer for rare disease diagnosis AI technology isn’t designed to replace clinicians - its function is to help them.
News FDA sets new decision date for Ipsen’s FOP therapy US agency sets 23rd August deadline for palovarotene review as Ipsen files more clinical data.
Digital Leave no one behind: Digital user research in rare disease Digital solutions have the potential to bridge care gaps for rare disease patients.
Patients Zebras, not horses: combatting rare disease via education Elsevier launches open-access journal 'Rare' and short-form videos to educate on rare disease.
Sales & Marketing Delivering for the 5 in 10,000 patients with a rare disease An estimated 30 million people are living with a rare disease in the European Union alone.
News Setback for MSD as German court blocks Keytruda SC launch A court in Germany has granted an injunction that will prevent MSD from distributing the new, subcutaneous version of its cancer drug Keytruda.
Sales & Marketing Sponsored How to meet your physicians where they are online A pharma company’s core mission is to improve patient outcomes. This hinges on effectively influencing HCP clinical behaviour and driving disease education.
