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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)central nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingPhase 32026-08-18NCT07221669
A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, congenital myasthenic syndrome, hereditary disease, nervous system disorder, neuromuscular disease, neuromuscular junction diseaseActive, not recruitingPhase 12026-07-24NCT06436742
A Hybrid Effectiveness-implementation Trial to Reduce Diabetes Distress in TeenagersNutritional and Metabolic Diseases, autoimmune disease, diabetes mellitus, endocrine system disorder, glucose metabolism disease, immune system disorder, metabolic disease, type 1 diabetes mellitusRecruitingNot applicable2026-05-29NCT06709755
Open-Label Study of mRNA-3927 in Participants With Propionic AcidemiaAcid-Base Imbalance, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, acidosis disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, propionic acidemiaRecruitingPhase 1 / Phase 22026-01-22NCT04159103
Soy Isoflavones For Inner City Infants At Risk For Asthma (SIRA) StudyPathological Conditions, Signs and Symptoms, Respiratory Sounds, Signs and Symptoms, Signs and Symptoms, Respiratory, WheezingRecruitingPhase 22025-11-21NCT05667701