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.
R&D LSX2026: On a therapeutic vision for ocular disease, with Al... Alexander Gebauer, co-founder & executive chairman of Galimedix Therapeutics, discusses transforming treatment of major neurodegenerative eye 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.
