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Molecular Genetics and Metabolism|August 24, 2019
Long-term comparative effectiveness of pegvaliase versus standard of care comparators in adults with phenylketonuriaRoberto Zori, Kirsten Ahring, Barbara Burton, et al.Molecular Genetics and Metabolism|July 29, 2011
Cystatin C and NT-proBNP as prognostic biomarkers in Fabry diseaseMiguel-Ángel Torralba-Cabeza, Susana Olivera, Derralynn A Hughes, et al.JIMD Reports|January 3, 2018
Two Uneventful Pregnancies in a Woman with Glutaric Aciduria Type 1Karolina M Stepien, Gregory M Pastores, Una Hendroff, et al.Journal of Bone and Mineral Research : the Official Journal of the American Society for Bone and Mineral Research|October 13, 2006
Effect of enzyme replacement therapy with imiglucerase on BMD in type 1 Gaucher diseaseRichard J Wenstrup, Katherine A Kacena, Paige Kaplan, et al.Metabolic Brain Disease|August 22, 2009
Multiple sulfatase deficiency: clinical report and description of two novel mutations in a Brazilian patientOsvaldo Alfonso Artigalás, Luiz Roberto da Silva, Maira Burin, et al.Blood|December 16, 2014
Type II NKT-TFH cells against Gaucher lipids regulate B-cell immunity and inflammationShiny Nair, Chandra Sekhar Boddupalli, Rakesh Verma, et al.Molecular Genetics and Metabolism|September 12, 2016
Home infusion of intravenous velaglucerase alfa: Experience from pooled clinical studies in 104 patients with type 1 Gaucher diseaseDeborah Elstein, T Andrew Burrow, Joel Charrow, et al.Journal of Inherited Metabolic Disease|December 1, 2011
Design, baseline characteristics, and early findings of the MPS VI (mucopolysaccharidosis VI) Clinical Surveillance Program (CSP)Christian J Hendriksz, Roberto Giugliani, Paul Harmatz, et al.Blood Cells, Molecules & Diseases|June 11, 2016
Development of anti-velaglucerase alfa antibodies in clinical trial-treated patients with Gaucher diseaseGregory M Pastores, Hadhami Ben Turkia, Derlis E Gonzalez, et al.Seminars in Hematology|October 7, 2004
Therapeutic goals in the treatment of Gaucher diseaseGregory M Pastores, Neal J Weinreb, Hans Aerts, et al.Pageof 10