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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular AtrophyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Atrophy, Spinal, Type II, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Spinal Muscular Atrophies of Childhood, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophy, spinal muscular atrophy, type IIIRecruitingPhase 22026-10-02NCT07047144
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioiCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Juvenile Spinal Muscular Atrophy, Muscular Atrophy, Spinal, Type II, Spinal Muscular Atrophies of Childhood, Spinal Muscular Atrophy Type I, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophyActive, not recruitingPhase 32026-09-24NCT04042025
GCB-001 in Treatment of Patients With Type II (SMA) Spinal Muscular AtrophyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Atrophy, Spinal, Type II, Spinal Muscular Atrophies of Childhood, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyEnrolling by invitationNot applicable2026-09-11NCT06772402
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of ApitegromabAtrophy, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Atrophy, Spinal, Type II, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Spinal Muscular Atrophies of Childhood, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, muscular atrophy, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophy, spinal muscular atrophy, type IIIActive, not recruitingPhase 32026-09-01NCT05626855
Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper LimbCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Atrophy, Spinal, Type II, Spinal Muscular Atrophies of Childhood, Spinal muscular atrophy 4, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophy, spinal muscular atrophy, type IIIActive, not recruitingNot applicable2025-08-26NCT06300996
Spinraza in Adult Spinal Muscular AtrophyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Atrophy, Spinal, Type II, Spinal Muscular Atrophies of Childhood, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy, spinal muscular atrophy, type IIIActive, not recruitingNot stated2024-11-20NCT03709784