Enzyme replacement therapy compared with best supportive care for the treatment of Pompe Disease: a systematic review and network meta-analysis

Background Late-onset Pompe disease is a rare inherited genetic condition that causes progressive muscle dysfunction and damage. As the disease advances, the progressive weakening of respiratory muscles significantly increases the risk of respiratory failure, which is a major contributor to prematur...

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Principais autores:	Mark Corbett, Chinyereugo Umemneku-Chikere, Sarah Nevitt, Nyanar Jasmine Deng, Matthew Walton, Helen Fulbright, Chong Yew Tan, Robin Lachmann, Rachel Churchill, Robert Hodgson
Format:	Artigo
Jezik:	Inglês
Izdano:	NIHR Journals Library 2026-02-01
Serija:	Health Technology Assessment
Teme:	glycogen storage disease type ii enzyme replacement therapy network meta-analysis humans
Online dostop:	https://doi.org/10.3310/GJRH0730
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