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Clinical trial condition

Myasthenic Syndromes, Congenital

Explore clinical trials studying Myasthenic Syndromes, Congenital. Study availability and eligibility vary by location and protocol.

11 public trials 4 recruiting studies
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Myasthenic Syndromes, Congenital trial results

Recruiting

A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4

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
View all 32 locations
View Trial Details
Recruiting

Congenital Muscle Disease Study of Patient and Family Reported Medical Information

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
  • Congenital Muscle Disease International Registry (www.cmdir.org) Lakewood, California, United States
View Trial Details
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
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Completed

Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes

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
View Trial Details

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