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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapycentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingPhase 42026-09-10NCT05861999
RZ358 Treatment for Congenital HyperinsulinismCongenital Hyperinsulinism, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, Nutritional and Metabolic Diseases, glucose metabolism disease, hyperinsulinism, hypoglycemia, metabolic disease, pancreas disorderActive, not recruitingPhase 32026-01-08NCT06208215