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Learn About Congenital Myasthenic Syndrome

What is the definition of Congenital Myasthenic Syndrome?
Congenital myasthenic syndromes (CMS) are a group of genetic conditions that all include muscle weakness that gets worse with physical activity. There are many subtypes of CMS with different symptoms, severity, and treatments. Symptoms range from mild to severe muscle weakness and may get worse over time or only occur periodically. The muscles of the face, neck, throat, eyes and limbs are most affected. There are at least 32 genes associated with CMS. Most are inherited in an autosomal recessive pattern. Genetic testing is necessary to tell the difference between subtypes.
What are the alternative names for Congenital Myasthenic Syndrome?
  • Congenital myasthenic syndromes
  • CMS
  • Congenital Myasthenia
  • Congenital myasthenic syndrome
Who are the top Congenital Myasthenic Syndrome Local Doctors?
Elite in Congenital Myasthenic Syndrome
Elite in Congenital Myasthenic Syndrome
Central Parkway, 
Newcastle Upon Tyne, ENG, GB 

Hanns Lochmuller practices in Newcastle Upon Tyne, United Kingdom. Mr. Lochmuller is rated as an Elite expert by MediFind in the treatment of Congenital Myasthenic Syndrome. His top areas of expertise are Congenital Myasthenic Syndrome, Myotonic Dystrophy, Myotonic Dystrophy Type 2, and Paramyotonia Congenita.

Elite in Congenital Myasthenic Syndrome
Elite in Congenital Myasthenic Syndrome

Regents Of The Univ Of Ca

2315 Stockton Blvd, 
Sacramento, CA 
Languages Spoken:
English

Ricardo Maselli is a Neurologist in Sacramento, California. Dr. Maselli is rated as an Elite provider by MediFind in the treatment of Congenital Myasthenic Syndrome. His top areas of expertise are Congenital Myasthenic Syndrome, Amyotonia Congenita, Lambert-Eaton Syndrome, and ADULT Syndrome.

 
 
 
 
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Elite in Congenital Myasthenic Syndrome
Pediatric Neurology | Pediatrics | Neurology
Elite in Congenital Myasthenic Syndrome
Pediatric Neurology | Pediatrics | Neurology

Mayo Clinic

200 1st St Sw, 
Rochester, MN 
Languages Spoken:
English
Accepting New Patients

Duygu Selcen is a Pediatric Neurologist and a Pediatrics provider in Rochester, Minnesota. Dr. Selcen is rated as an Elite provider by MediFind in the treatment of Congenital Myasthenic Syndrome. Her top areas of expertise are Congenital Myasthenic Syndrome, Amyotonia Congenita, Tubular Aggregate Myopathy, and Congenital Fiber-Type Disproportion. Dr. Selcen is currently accepting new patients.

What are the latest Congenital Myasthenic Syndrome Clinical Trials?
Multicenter, Multinational, Natural History Study in Participants With Congenital Myasthenic Syndromes Due to Mutations in DOK7, MUSK, AGRN, or LRP4

Summary: Participants will attend up to 4 study visits to collect clinical assessments. The assessments will evaluate participants' symptoms and quality of life to understand disease activity in patients with CMS due to mutations in DOK7, MUSK, AGRN, or LRP4.

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A Single Center Prospective Natural History and Outcome Measure Validation Study of Congenital Myasthenic Syndromes

Background: Congenital myasthenic syndromes (CMSs) are a group of inherited disorders that affect how the nerves communicate with muscles. These can cause many problems that affect how people can move and use their bodies.

Who are the sources who wrote this article ?

Published Date: May 02, 2022
Published By: Genetic and Rare Diseases Informnation Center