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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, Myotonia, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular diseaseActive, not recruitingPhase 1 / Phase 22026-08-20NCT06185764
Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)Congenital, 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 dystrophy type 1, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular diseaseRecruitingNot stated2026-06-10NCT03981575
Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatmentscentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingNot stated2023-03-14NCT05768048
Natural History of SMAcentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingNot stated2023-03-06NCT05755451