An Atypical Case of Head Tremor and Extensive White Matter in an Adult Female Caused by 3-Hydroxy-3-methylglutaryl-CoA Lyase Deficiency.
Nassim BoutouchentJulie BourilhonBénédicte Sudrié-ArnaudAntoine BonnevalleLucie Guyant-MaréchalCécile Acquaviva BourdainLoréna Dujardin-IppolitoSoumeya BekriIvana DabajAbdellah TebaniPublished in: Diagnostics (Basel, Switzerland) (2021)
3-Hydroxy-3-methylglutaryl-CoA (HMG-CoA) Lyase deficiency (HMGLD) (OMIM 246450) is an autosomal recessive genetic disorder caused by homozygous or compound heterozygous variants in the HMGCL gene located on 1p36.11. Clinically, this disorder is characterized by a life-threatening metabolic intoxication with a presentation including severe hypoglycemia without ketosis, metabolic acidosis, hyper-ammoniemia, hepatomegaly and a coma. HMGLD clinical onset is within the first few months of life after a symptomatic free period. In nonacute periods, the treatment is based on a protein- and fat-restricted diet. L-carnitine supplementation is recommended. A late onset presentation has been described in very few cases, and only two adult cases have been reported. The present work aims to describe an incidental discovery of an HMGLD case in a 54-year-old patient and reports a comprehensive review of clinical and biological features in adult patients to raise awareness about the late-onset presentation of this disease.
Keyphrases
- late onset
- early onset
- case report
- fatty acid
- copy number
- white matter
- genome wide
- type diabetes
- replacement therapy
- small molecule
- physical activity
- parkinson disease
- emergency department
- gene expression
- young adults
- skeletal muscle
- intellectual disability
- protein protein
- metabolic syndrome
- autism spectrum disorder
- glycemic control
- amino acid