AP-HP Hopital Necker-Enfants Malades

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
Long-term Extension of GTX-102 in Angelman Syndrome	Abnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorder	Active, not recruiting	Phase 3	2026-09-09		NCT06415344
A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG	Dyskinesias, Neurologic Manifestations, PMM2-congenital disorder of glycosylation, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, atactic disorder, non-human animal, nervous system disorder	Active, not recruiting	Phase 2 / Phase 3	2026-08-14		NCT06892288

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