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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 SkippingCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Infant, Newborn, Diseases, Muscular Disorders, Atrophic, X-linked disease, congenital, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular diseaseRecruitingPhase 32026-09-29NCT07587242
A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHDCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, facioscapulohumeral muscular dystrophy, facioscapulohumeral muscular dystrophy 1, facioscapulohumeral muscular dystrophy 2, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular diseaseRecruitingPhase 32026-07-27NCT07038200
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, brain disorder, central nervous system disorder, disorder of glycogen metabolism, glycogen storage disease II, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, nervous system disorderRecruitingPhase 22026-06-23NCT07123155