Long-term follow-up of 17 patients with childhood Pompe disease treated with enzyme replacement therapy.

Jan C van der MeijdenMichelle E KruijshaarLaurike HarlaarDimitris RizopoulosNadine A M E van der BeekAns T van der Ploeg

Published in: Journal of inherited metabolic disease (2018)

We report a positive effect of ERT in patients with childhood Pompe disease at group level. For some patients, new or personalized treatments should be considered.

Keyphrases

replacement therapy
smoking cessation
late onset
newly diagnosed
end stage renal disease
ejection fraction
chronic kidney disease
prognostic factors
early life
childhood cancer
young adults
patient reported outcomes