Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD	Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, facioscapulohumeral muscular dystrophy, facioscapulohumeral muscular dystrophy 1, facioscapulohumeral muscular dystrophy 2, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease	Recruiting	Phase 3	2026-07-27		NCT07038200
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 disorder	Recruiting	Phase 2	2026-06-23		NCT07123155

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