Literature DB >> 31580529

Two middle-aged women with the Finnish variant of muscle-eye-brain disease (MEB).

Maria Arvio1,2,3,4, Laura Määttänen5, Maria Haanpää2, Jaana Lähdetie6.   

Abstract

Muscle-eye-brain disease (MEB) is a recessively inherited rare disease. Sixteen different gene mutations are known, with the most common mutations in the POMGNT1 gene. The disease is now called congenital muscular dystrophy-dystroglycanopathy type A3 (MDDGA3). It manifests itself as muscular dystrophy with eye and brain anomalies and intellectual disability. Previous clinical reports describe young patients. We have been able to follow two patients for almost 40 years. Their clinical picture has remained quite stable since adolescence, appearing as severe intellectual and motor disability, extremely limited communication skills, visual impairment, epilepsy, joint contractures, repeated bowel obstructions, teeth abrasion due to bruxism, an irregular sleep pattern and as a previously unreported feature hypothermic periods manifesting as excessive sleepiness.
© 2019 Wiley Periodicals, Inc.

Entities:  

Keywords:  zzm321990POMGNT1 gene; MEB; dystroglycanopathia; muscle-eye-brain disease

Year:  2019        PMID: 31580529     DOI: 10.1002/ajmg.a.61369

Source DB:  PubMed          Journal:  Am J Med Genet A        ISSN: 1552-4825            Impact factor:   2.802


  1 in total

Review 1.  Clinical and Molecular Spectrum of Muscular Dystrophies (MDs) with Intellectual Disability (ID): a Comprehensive Overview.

Authors:  Malihe Mohamadian; Mandana Rastegar; Negin Pasamanesh; Ata Ghadiri; Pegah Ghandil; Mohsen Naseri
Journal:  J Mol Neurosci       Date:  2021-11-02       Impact factor: 3.444

  1 in total

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