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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman SyndromeAbnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorderActive, not recruitingPhase 32026-09-29NCT06914609
A Study to Evaluate the Efficacy and Safety of KarXT for the Treatment of Manic Episodes in Bipolar-I DisorderMania, Neurobehavioral Manifestations, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, nervous system disorderRecruitingPhase 32026-09-22NCT06951698
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)Congenital, 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 diseaseActive, not recruitingPhase 22026-09-15NCT05996003
A Study to Assess the Long-term Safety of KarXT for the Treatment of Manic Episodes in Bipolar-I Disorder (BALSAM-3)Mania, Neurobehavioral Manifestations, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, nervous system disorderRecruitingPhase 32026-09-14NCT06929273
A Study to Evaluate the Efficacy and Safety of KarXT in Acutely Psychotic Japanese Adult Participants With SchizophreniaSchizophrenia Spectrum and Other Psychotic Disorders, psychiatric disorder, schizophreniaRecruitingPhase 32026-07-27NCT06882785
Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)Behcet disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Hemic and Lymphatic Diseases, Hemorrhage, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Purpura, Thrombocytopenic, Idiopathic, Signs and Symptoms, Skin Manifestations, Skin and Connective Tissue Diseases, Stomatognathic Diseases, anterior uveitis, antiphospholipid syndrome, autoimmune disease, blood coagulation disease, blood platelet disease, bullous pemphigoid, cardiovascular disorder, connective tissue disorder, dermatomyositis, eye disorder, hematologic disorder, hemorrhagic disease, hereditary disease, hereditary periodic fever syndrome, hereditary skin disorder, immune system disorder, mouth disorder, muscle tissue disorder, musculoskeletal system disorder, myositis disease, nervous system disorder, neuromuscular disease, panuveitis, polymyositis, purpura, skin disorder, skin vascular disease, thrombocytopenia, thrombocytopenic purpura, thrombotic microangiopathy, uveal disorder, uveitis, vascular disorder, vasculitis, vesiculobullous skin diseaseRecruitingPhase 12026-07-22NCT06371417
Phase 1b Long-term Extension Trial of RAY121 in Immunological Diseases (RAINBOW-LTE Trial)Behcet disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Hemic and Lymphatic Diseases, Hemorrhage, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Purpura, Thrombocytopenic, Idiopathic, Signs and Symptoms, Skin Manifestations, Skin and Connective Tissue Diseases, Stomatognathic Diseases, anterior uveitis, antiphospholipid syndrome, autoimmune disease, blood coagulation disease, blood platelet disease, bullous pemphigoid, cardiovascular disorder, connective tissue disorder, dermatomyositis, eye disorder, hematologic disorder, hemorrhagic disease, hereditary disease, hereditary periodic fever syndrome, hereditary skin disorder, immune system disorder, mouth disorder, muscle tissue disorder, musculoskeletal system disorder, myositis disease, nervous system disorder, neuromuscular disease, panuveitis, polymyositis, purpura, skin disorder, skin vascular disease, thrombocytopenia, thrombocytopenic purpura, thrombotic microangiopathy, uveal disorder, uveitis, vascular disorder, vasculitis, vesiculobullous skin diseaseEnrolling by invitationPhase 12026-07-17NCT06723106
NS-050/NCNP-03 in Boys With DMD (Meteor50)Congenital, 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 diseaseActive, not recruitingPhase 1 / Phase 22026-06-16NCT06053814
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)Congenital, 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 diseaseActive, not recruitingPhase 32026-05-22NCT05881408
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical StudyCongenital, 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 diseaseEnrolling by invitationPhase 32026-02-06NCT05967351
International Registry Study of Neutral Lipid Storage Disease (NLSD) / Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Related DiseasesDorfman-Chanarin disease, Neutral Lipid Storage Disease with Myopathy, neutral lipid storage diseaseRecruitingNot stated2026-02-05NCT02918032
Study of the Efficacy and Safety for Rituximab in Myalgia Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS)Chronic Disease, Disease Attributes, Neuroinflammatory Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, encephalomyelitis, muscle tissue disorder, musculoskeletal system disorder, myalgic encephalomeyelitis/chronic fatigue syndrome, nervous system disorder, neuromuscular diseaseRecruitingPhase 22025-04-30NCT06952413