Sudden deterioration in nonclassical infantile-onset Pompe disease responding to alglucosidase alfa infusion therapy: a case report

J Inherit Metab Dis. 2006 Dec;29(6):763. doi: 10.1007/s10545-006-0427-4. Epub 2006 Oct 14.

Abstract

A patient with atypical infantile Pompe disease suffered acute respiratory insufficiency at the age of 8 years which resulted in complete immobilization and dependence on assisted ventilation. Shortly after initiation of enzyme replacement therapy, she regained her mobility and, after 20 months of treatment, she now leads an almost normal life with limited restrictions.

Publication types

  • Case Reports

MeSH terms

  • Child
  • Female
  • Glycogen Storage Disease Type II / drug therapy*
  • Humans
  • Respiratory Insufficiency / drug therapy*
  • Time Factors
  • Treatment Outcome
  • alpha-Glucosidases / therapeutic use*

Substances

  • GAA protein, human
  • alpha-Glucosidases