Enzyme therapy for Pompe disease with recombinant human α‐glucosidase from rabbit milk
- 1 April 2001
- journal article
- clinical trial
- Published by Wiley in Journal of Inherited Metabolic Disease
- Vol. 24 (2) , 266-274
- https://doi.org/10.1023/a:1010383421286
Abstract
Pompe disease is a metabolic myopathy caused by deficiency of lysosomal acid α-glucosidase. In this report we review the first 36 weeks of a clinical study on the safety and efficacy of enzyme...Keywords
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