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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
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
UK SMA Patient RegistryCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Kennedy disease, Spinal Muscular Atrophies of Childhood, X-linked disease, autosomal recessive distal spinal muscular atrophy 1, 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 atrophyRecruitingNot stated2026-08-20NCT04292574
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophycentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophyActive, not recruitingPhase 32026-06-01NCT05337553
Development of Non-Invasive Prenatal Diagnosis for Single Gene DisordersAbnormalities, Multiple, Becker muscular dystrophy, Ciliopathies, Cognition Disorders, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Duchenne muscular dystrophy, Dyskinesias, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Huntington disease, Infant, Newborn, Diseases, Male Urogenital Diseases, Muscular Disorders, Atrophic, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, Neurobehavioral Manifestations, Neurologic Manifestations, Polycystic Kidney, Autosomal Recessive, Sex Chromosome Disorders, Urogenital Diseases, X-linked disease, X-linked hydrocephalus with stenosis of the aqueduct of Sylvius, anemia, basal ganglia disorder, blood coagulation disease, brain disorder, central nervous system disorder, choreatic disease, chromosomal disorder, coagulation protein disease, cognitive disorder, cystic fibrosis, cystic kidney disease, dementia, familial hemolytic anemia, fragile X syndrome, hematologic disorder, hemolytic anemia, hemophilia A, hemophilia B, hemorrhagic disease, hereditary disease, hereditary neoplastic syndrome, inherited blood coagulation disorder, inherited hemoglobinopathy, inherited neurodegenerative disorder, intellectual disability, kidney disorder, lung disorder, movement disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis-Noonan syndrome, neuromuscular disease, pancreas disorder, polycystic kidney disease, proximal spinal muscular atrophy, psychiatric disorder, respiratory system disorder, sickle cell disease, urinary system disorderRecruitingNot stated2026-04-23NCT06147414
Swiss Registry for Neuromuscular DisordersDuchenne muscular dystrophy, congenital muscular dystrophy, proximal spinal muscular atrophyRecruitingNot stated2026-01-15NCT05102916
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
A Head-to-head Study Comparing the Functional Value of Two Models of Robotically Assisted Rehabilitation in SMA (Spinal Muscular Atrophy) Patientsproximal spinal muscular atrophyRecruitingNot applicable2024-03-21NCT06322654