congenital myasthenic syndrome
congenital myasthenic syndrome
Explore linked clinical trials. Standard reference: MONDO:00189407 results ยท Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, congenital myasthenic syndrome, hereditary disease, nervous system disorder, neuromuscular disease, neuromuscular junction disease | Recruiting | Not stated | 2026-09-25 | NCT06078553 | |
| Evaluating the Safety and Effects of AMP-101 in People With DOK7 Congenital Myasthenia Syndrome | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, congenital myasthenic syndrome, hereditary disease, nervous system disorder, neuromuscular disease, neuromuscular junction disease | Not yet recruiting | Phase 1 | 2026-09-21 | NCT07830563 | |
| A Prospective Natural History and Outcome Measure Validation Study of Congenital Myasthenic Syndromes | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, congenital myasthenic syndrome, hereditary disease, nervous system disorder, neuromuscular disease, neuromuscular junction disease | Recruiting | Not stated | 2026-08-13 | NCT06630650 | |
| A Study to Assess Efficacy and Safety of Adimanebart in Adult and Pediatric Participants With DOK7-,MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, congenital myasthenic syndrome, hereditary disease, nervous system disorder, neuromuscular disease, neuromuscular junction disease | Not yet recruiting | Phase 3 | 2026-08-04 | NCT07746089 | |
| 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 disease | Active, not recruiting | Phase 1 | 2026-07-24 | NCT06436742 | |
| Patients With Congenital Myasthenic Syndrome Will be Treated With Mesenchymal Stem Cell Exosome Solution | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, congenital myasthenic syndrome, hereditary disease, nervous system disorder, neuromuscular disease, neuromuscular junction disease | Recruiting | Phase 1 | 2025-11-10 | NCT07226726 | |
| Congenital Muscle Disease Study of Patient and Family Reported Medical Information | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Emery-Dreifuss muscular dystrophy, Eye Diseases, Hereditary, Malformations of Cortical Development, Group II, Muscular Disorders, Atrophic, Neurobehavioral Manifestations, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Thomsen and Becker disease, Wieacker-Wolff syndrome, X-linked disease, autosomal recessive limb-girdle muscular dystrophy type 2G, autosomal recessive limb-girdle muscular dystrophy type 2M, brain disorder, central nervous system disorder, central nervous system malformation, cerebral cortical dysplasia, cobblestone lissencephaly, congenital muscular dystrophy due to integrin alpha-7 deficiency, congenital myasthenic syndrome, epilepsy, eye disorder, hereditary disease, inherited neurodegenerative disorder, intellectual disability, limb-girdle muscular dystrophy, lissencephaly spectrum disorders, muscle tissue disorder, muscular dystrophy, muscular dystrophy-dystroglycanopathy, type A, musculoskeletal system disorder, myotonic syndrome, nervous system disorder, neurodegenerative disease, neurodevelopmental disorder, neuromuscular disease, neuromuscular junction disease, psychiatric disorder | Recruiting | Not stated | 2021-08-09 | NCT01403402 |