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c.754T>A homozygous mutation described for the first time in three Moroccan patients with Gaucher disease.

Ghizlane ZouiriHajar RhoudaYamna Kriouile
Published in: Archives de pediatrie : organe officiel de la Societe francaise de pediatrie (2024)
Gaucher disease (GD) is a lysosomal storage disorder caused by glucocerebrosidase (GBA) deficiency. There are three subcategories of GD: Type 1 is characterized by the absence of primary central nervous system involvement; type 2 is an acute neuropathic disorder; and type 3 is chronic neuropathic. The correlation between genotype and phenotype is sometimes difficult to establish. The F213I (c.754T>A p.Phe252Ile) mutation was reported to be a unique mutation in Asia. To our knowledge, this is the first time the c.754T>A p.(Phe252Ile) mutation (homozygous state) is reported in a Moroccan population and is associated with GD type 2 (two patients) and GD type 3 (one patient).
Keyphrases
  • end stage renal disease
  • healthcare
  • chronic kidney disease
  • replacement therapy
  • newly diagnosed
  • ejection fraction
  • liver failure
  • drug induced
  • prognostic factors
  • mechanical ventilation