Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease: a clinical study and review of the literature.

Aleena A KhanLaura E CaseMrudu HerbertStephanie DeArmeyHarrison JonesKelly CrispKanecia ZimmermanMai K ElMallahSarah P YoungPriya S Kishnani

Published in: Genetics in medicine : official journal of the American College of Medical Genetics (2020)

Higher rhGAA doses are safe, improve gross motor outcomes, lingual strength, pulmonary function measures, and biochemical markers in early-onset Pompe disease, and should be considered in patients with clinical and functional decline.

Keyphrases

late onset
early onset
replacement therapy
type diabetes
metabolic syndrome
glycemic control
insulin resistance
double blind
children with cerebral palsy