| Literature DB >> 31580529 |
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.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