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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 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
Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular DisordersCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, Spinal Muscular Atrophies of Childhood, X-linked disease, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy, spinal muscular atrophy, type IIIRecruitingNot stated2026-04-09NCT06839469
Gait and Bone Health in SMACongenital, Hereditary, and Neonatal Diseases and Abnormalities, Spinal Muscular Atrophies of Childhood, bone fracture, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, injury, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy, spinal muscular atrophy, type IIIRecruitingNot stated2026-03-18NCT07400198
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
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 PatientsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, 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 IIIRecruitingPhase 1 / Phase 22025-07-03NCT06421831
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