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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508delCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderActive, not recruitingPhase 32026-10-09NCT05331183
Study of SRP-1003 in Participants With Type 1 Myotonic DystrophyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular diseaseEnrolling by invitationPhase 1 / Phase 22026-10-05NCT06138743
GARDE : GC7 (N1-guanyl-1,7 Diaminoheptane) cARDiac arrEstcardiac arrest, cardiovascular disorder, heart disorderNot yet recruitingNot applicable2026-03-16NCT07468292