Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH
Barbara A. Konkle, Glen Pierce, Donna Coffin, Mayss Naccache, R. Cary Clark, Lindsey A. George, Alfonso Iorio, Brian O’Mahony, Steven W. Pipe, Mark W. Skinner, Crystal Watson, Flora Peyvandi, Johnny Mahlangu
Topics & Concepts
MedicineClinical trialObservational studyQuality of life (healthcare)Intensive care medicineFamily medicineInternal medicineNursingHemophilia Treatment and ResearchVirus-based gene therapy researchNeurogenetic and Muscular Disorders Research