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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, Myotonia, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, hereditary disease, inherited neurodegenerative disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myotonic dystrophy, myotonic syndrome, nervous system disorder, neurodegenerative disease, neuromuscular diseaseActive, not recruitingPhase 1 / Phase 22026-08-20NCT06185764
Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, brain disorder, central nervous system disorder, disorder of glycogen metabolism, glycogen storage disease II, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, nervous system disorderRecruitingPhase 22026-06-23NCT07123155
Understanding the Variation of Modern Endoscopic Ultrasound Use in Patients With Oesophageal Cancer (VALUE)Digestive System Diseases, Digestive System Neoplasms, Esophageal Neoplasms, Head and Neck Neoplasms, Neoplasms by Site, digestive system disorder, digestive system neoplasm, esophageal disorder, neoplasmRecruitingNot stated2026-04-30NCT06440174
Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Gaucher Disease, Type 1, Gaucher disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosisActive, not recruitingPhase 1 / Phase 22025-11-04NCT06545136
Ambroxol to Slow Progression in Parkinson DiseaseNutritional and Metabolic Diseases, Parkinson disease, basal ganglia disorder, brain disorder, central nervous system disorder, metabolic disease, movement disorder, nervous system disorder, neurodegenerative disease, parkinsonian disorder, proteostasis deficiencies, synucleinopathyRecruitingPhase 32025-05-01NCT05778617
The Role of Ixazomib in Autologous Stem Cell Transplant in Relapsed Myeloma - Myeloma XII (ACCoRd)Blood Protein Disorders, Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, cardiovascular disorder, hematologic disorder, hemorrhagic disease, immune system disorder, immunoproliferative disorder, lymphoproliferative syndrome, monoclonal gammopathy, neoplasm, plasma cell neoplasm, vascular disorder, vascular hemostatic diseaseRecruitingPhase 32018-06-19NCT03562169