The Natural Course of Infantile Pompe’s Disease: 20 Original Cases Compared With 133 Cases From the Literature

M.P. van den Hout, Hannerieke; Hop, Wim; P. van Diggelen, Otto; Smeıtınk, Jan; Smit, Gerrit; Tien Poll‐The, Bwee; D. Bakker, H.; J. J. E. Loonen, Maarten; B.C. de Klerk, Johannis; Reuser, Arnold; T. van der Ploeg, Ans

doi:10.1542/peds.112.2.332

Public

The Natural Course of Infantile Pompe’s Disease: 20 Original Cases Compared With 133 Cases From the Literature

Published in PEDIATRICS • Aug 1, 2003

Authors:

Hannerieke M.P. van den Hout

Wim Hop

Otto P. van Diggelen

Abstract

OBJECTIVE: Infantile Pompe's disease is a lethal cardiac and muscular disorder. Current developments toward enzyme replacement therapy are promising. The aim of our study is to delineate the natural course of the disease to verify endpoints of clinical studies. METHODS: A total of 20 infantile patie...

Subject

Medicine

Creatine kinase

Enzyme replacement therapy

Research Assistant

AI chat, annotations, notes & similar papers

Finding related papers...

Discussions

(0)

No comments yet

Be the first to share your thoughts!