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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
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
An Open-Label Extension Study of BPN14770 in Subjects With Fragile X SyndromeCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Sex Chromosome Disorders, X-linked disease, chromosomal disorder, fragile X syndrome, hereditary disease, inherited neurodegenerative disorder, intellectual disability, nervous system disorderActive, not recruitingPhase 32025-10-03NCT05367960