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NCT06078553
- Conditions
- Congenital Myasthenic Syndrome, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +4 more
- Locations
-
- UC Davis Health - UC Davis Health Midtown Ambulatory Care Center Sacramento, California, United States
- Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital Chicago, Illinois, United States
- Hospital Sisters Health System (HSHS) - St Elizabeth's Hospital O'Fallon, Illinois, United States
- Harvard Medical School - Boston Children's Hospital Boston, Massachusetts, United States
NCT06630650
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Myasthenic Syndromes, Congenital, +3 more
- Locations
-
- National Institutes of Health Clinical Center Bethesda, Maryland, United States
NCT07226726
- Conditions
- Congenital Myasthenic Syndrome, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +4 more
- Locations
-
- Prodromos Stem Cell Institute Naples, Florida, United States
- Prodromos Stem Cell Institute Irving, Texas, United States
- Medical Surgical Associates Center St John's, Antigua and Barbuda
NCT01403402
- Conditions
- Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Fatty Liver and Infantile-onset Cataract Caused by TRAPPC11 Mutations), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan and Epilepsy), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan), +89 more
- Locations
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- Congenital Muscle Disease International Registry (www.cmdir.org) Lakewood, California, United States
Completed
Patients With ALS and Other Motor Disorders Will be Treated With Mesenchymal Cell Exosome Solution
NCT07105371
- Conditions
- Basal Ganglia Diseases, Brain Diseases, Bulbo-Spinal Atrophy, X-Linked, +21 more
- Locations
-
- Medical Surgical Associates Center St John's, Antigua and Barbuda
Completed
Baby Detect : Genomic Newborn Screening
NCT05687474
- Conditions
- 3-Hydroxy 3-Methyl Glutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency, +368 more
- Locations
-
- CRMN, Hôpital La Citadelle Liège, Wallonia, Belgium
NCT05408702
- Conditions
- Autoimmune Diseases, Autoimmune Diseases of the Nervous System, Autoimmune Myasthenia Gravis, +16 more
- Locations
-
- Online Questionnaire Paris, France
NCT02562066
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Myasthenic Syndromes, Congenital, +3 more
- Locations
-
- UCLA Department of Neurology Los Angeles, California, United States
- Johns Hopkins Pediatric Neurology Baltimore, Maryland, United States
- Boston Children's Hospital Boston, Massachusetts, United States
- Ohio State University Columbus, Ohio, United States
NCT01203592
- Conditions
- Congenital Myasthenic Syndrome, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +4 more
- Locations
-
- Mayo Clinic Rochester, Minnesota, United States
NCT01474980
- Conditions
- Congenital Myasthenic Syndrome, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +4 more
- Locations
-
Not specified
Active, Not Recruiting
A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)
NCT06436742
- Conditions
- CMS, Congenital Myasthenic Syndrome, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +5 more
- Locations
-
- UC Davis Medical Center Sacramento, California, United States
- Ann and Robert H Lurie Childrens Hospital of Chicago Chicago, Illinois, United States
- Ottawa Hospital Research Institute - Civic Campus Ottawa, Canada
- CHU - Hospital de la Timone Marseille, France