Latest Advances

  • Condition: Pompe disease
  • Journal: Genetics in medicine : official journal of the American College of Medical Genetics
  • Treatment Used: 4000 liter scale alglucosidase
  • Number of Patients: 113
  • Published —
The purpose of the study was to evaluate 4,000 L alglucosidase alfa in US infantile- or late-onset Pompe disease patients up to 1 year old.
  • Condition: CRIM-Negative Infantile Pompe Disease
  • Journal: Molecular genetics and metabolism
  • Treatment Used: High Dose Intravenous Immunoglobulin (IVIG)
  • Number of Patients: 1
  • Published —
This case report describes a a 7-year-old patient with cross-reactive immunologic material (CRIM)-negative infantile Pompe disease (IPD) treated with high dose and high frequency intravenous immunoglobulin (IVIG).
Showing 1-2 of 2