[Bardet Biedl syndrome: a case report].
Nuria GrimbergMaría E AndrésMabel FerraroPublished in: Archivos argentinos de pediatria (2022)
Bardet Biedl syndrome is an autosomal recessive ciliopathie. It is a pleiotropic disorder characterised by retinal dystrophy, renal dysfunction, polydactyly, obesity, cognitive deficit and hypogenitalism. Diagnosis is based on clinical features. Molecular genetic testing is available. There is no specific treatment, a multidisciplinary approach is required. We report the case of a 13-year-old female patient with obesity and hyperphagia, type 2 diabetes, hypothyroidism, polydactyly, cognitive deficit and visual impairment. A multigenic panel allowed the identification of two heterozygous pathogenic variants in the BBS2 gene.
Keyphrases
- type diabetes
- insulin resistance
- case report
- metabolic syndrome
- weight loss
- copy number
- early onset
- high fat diet induced
- weight gain
- cardiovascular disease
- glycemic control
- optical coherence tomography
- oxidative stress
- diabetic retinopathy
- skeletal muscle
- replacement therapy
- gene expression
- body mass index
- quality improvement
- autism spectrum disorder
- transcription factor
- duchenne muscular dystrophy