R&D Drug development and working with specialised Rare Disease n... There are currently an estimated 300 million people living with one or more Rare Diseases across the world.
Webinars Sponsored The power of R&D collaborations to improve outcomes in rare ... Far more common than initially thought, rare disease treatments remain far too scarce.
Views & Analysis How does HTA for orphan drugs differ across Europe? New research looks at the factors that speed up and slow down HTA appraisals for rare disease medicines across Europe.
Views & Analysis The biggest challenges facing rare disease pharma – and how ... As Rare Disease day 2020 approaches, we take a look at the biggest challenges facing orphan drug developers and ask whether the future is bright or bleak for these difficult conditions.
Partner Content Partner Content Exclusive Bionical EMAS speaker interview released for Orpha... Exclusive Bionical EMAS speaker interview released
Views & Analysis Who will be the biggest pharma players in orphan drugs? New analysis from GlobalData has identified the predicted top 15 pharma players in 2025 for organ drugs, with Roche coming out in the lead.
News Neurogene drops high-dose arm in Rett study after death The patient affected by a serious adverse event in Neurogene's clinical trial of Rett syndrome gene therapy has now died
Sales & Marketing Coming soon: The Life Sciences Industry Report 2025 – insigh... Prepare for 2025 with pharmaphorum’s Life Sciences Report, featuring expert insights on AI in drug development, gene therapy, and top industry trends
