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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR GeneX-Linked Retinitis PigmentosaActive, not recruitingPhase 32026-09-25NCT04794101
A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and BreastfeedingBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases and Pregnancy Complications, Nutritional and Metabolic Diseases, Urogenital Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, maternal phenylketonuria, metabolic disease, nervous system disorder, phenylketonuria, pregnancy disorderRecruitingNot stated2026-03-24NCT05579548