Algorithm for the Early Diagnosis and Treatment of Patients with Cross Reactive Immunologic Material-Negative Classic Infantile Pompe Disease: A Step towards Improving the Efficacy of ERT

Algorithm for the Early Diagnosis and Treatment of Patients with Cross Reactive Immunologic Material-Negative Classic Infantile Pompe Disease: A Step towards Improving the Efficacy of ERT

Objective Although enzyme replacement therapy (ERT) is a highly effective therapy, CRIM-negative (CN) infantile Pompe disease (IPD) patients typically mount a strong immune response which abrogates the efficacy of ERT, resulting in clinical decline and death. This study was designed to demonstrate t...

Повний опис

Збережено в:
Бібліографічні деталі
Автори: Suhrad G. Banugaria, Sean N. Prater, Trusha Patel, Stephanie DeArmey, Christie Milleson, Kathryn L. Berrier, Deeksha Bali, Catherine Rehder, Julian Raiman, Raymond A. Wang, François Labarthe, Joel Charrow, Paul Harmatz, Pranesh Chakraborty, Amy S. Rosenberg, Priya S. Kishnani
Формат: Artigo
Мова:Англійська
Опубліковано: 2013
Онлайн доступ:https://doi.org/10.1371/journal.pone.0067052
https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0067052&type=printable
Теги: Додати тег
Немає тегів, Будьте першим, хто поставить тег для цього запису!

Інтернет

https://doi.org/10.1371/journal.pone.0067052
https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0067052&type=printable

Схожі ресурси