Early initiation of enzyme replacement therapy in classical Fabry disease normalizes biomarkers in clinically asymptomatic pediatric patients
Author:
Funder
Sanofi
Publisher
Elsevier BV
Subject
Endocrinology,Genetics,Molecular Biology
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4. Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 98 hemizygous males;MacDermot;J. Med. Genet.,2001
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