Clinical features and predictors for disease natural progression in adults with Pompe disease: a nationwide prospective observational study

Clinical features and predictors for disease natural progression in adults with Pompe disease: a nationwide prospective observational study

<p>Abstract</p> <p>Background</p> <p>Due partly to physicians’ unawareness, many adults with Pompe disease are diagnosed with great delay. Besides, it is not well known which factors influence the rate of disease progression, and thus disease outcome. We delineated the...

Full description

Bibliographic Details
Main Authors: van der Beek Nadine AME, de Vries Juna M, Hagemans Marloes LC, Hop Wim CJ, Kroos Marian A, Wokke John HJ, de Visser Marianne, van Engelen Baziel GM, Kuks Jan BM, van der Kooi Anneke J, Notermans Nicolette C, Faber Karin G, Verschuuren Jan JGM, Reuser Arnold JJ, van der Ploeg Ans T, van Doorn Pieter A
Format: Article
Language:English
Published: BMC 2012-11-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Acid α-glucosidase
Glycogen storage disease type II
OMIM number 232300
Lysosomal storage disorder
Disease progression
Natural course
Prognostic factors
Online Access:http://www.ojrd.com/content/7/1/88

Internet

http://www.ojrd.com/content/7/1/88

Similar Items