Avalglucosidase alfa in infantile-onset Pompe disease: A snapshot of real-world experience in Italy
Author:
Funder
Sanofi Genzyme
Publisher
Elsevier BV
Reference32 articles.
1. Pompe Disease: Glycogen Storage Disease type II, Acid Alpha-Glucosidase (Acid Maltase) Deficiency;Reuser,2018
2. How to describe the clinical spectrum in Pompe disease?;Gungor;Am. J. Med. Genet. A,2013
3. Motor responses in pediatric Pompe disease in the ADVANCE participant cohort;Duong;J. Neuromusc. Dis.,2022
4. Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study;van Gelder;J. Inherit. Metab. Dis.,2016
5. Response of 33 UK patients with infantile-onset Pompe disease to enzyme replacement therapy;Broomfield;J. Inherit. Metab. Dis.,2016
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