News Pharming brings first drug for rare disease APDS to US Joenja will launch in April and could make upwards of $200m a year in sales, say analysts.
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.
Spotlight on World Without Disease initiative: 2024 update In a new white paper from the World Without Disease initiative, a 2024 update is provided of the current endeavours and developments that have occurred since inaugural discussions last year
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