The humanistic burden of Pompe disease: are there still unmet needs? A systematic review.

Benedikt SchoserDeborah A BilderDavid DimmockDigant GuptaEmma S JamesSuyash Prasad

Published in: BMC neurology (2017)

Pompe disease has a substantial humanistic burden, with strong inter-relationships among and between humanistic burden parameters and clinical progression.

Keyphrases

late onset
replacement therapy
risk factors
early onset