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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Rescue of Nephrons With ALE.F02 (RENAL-F02)Glomerulonephritis Rapidly ProgressiveRecruitingPhase 22026-08-14NCT06047171
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular diseaseRecruitingPhase 1 / Phase 22026-08-13NCT07038824
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR VariantAlzheimer disease, Amyloid Neuropathies, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, amyloidosis, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cognitive disorder, dementia, familial amyloid neuropathy, heart disorder, hereditary amyloidosis, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, polyneuropathy, proteostasis deficiencies, psychiatric disorder, tauopathyRecruitingPhase 32026-08-04NCT06563895