New drug offers potential cure for ultra rare inherited condition A teenage patient who helped discover a rare condition is the first to benefit from a new treatment.

#rarediseases #ultrararediseases #immunology #Joenja #leniolisib #PharmingTechnologies #raredisease #ActivatedPI3kinaseDeltaSyndrome #APDS #UniversityofCambridge #Addenbrookes #AnitaChandra #disregulatedimmunesystem #APDSpatients #SpecialisedCommissioning
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This study used MCDA for Orphan Drugs to compare #leniolisib vs. standard of care in APDS. A panel found leniolisib superior in efficacy, safety, QoL, and cost, making it a valuable treatment.
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