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Molecular Genetics and Metabolism|June 6, 2015
Long-term velaglucerase alfa treatment in children with Gaucher disease type 1 naïve to enzyme replacement therapy or previously treated with imigluceraseLaurie Smith, William Rhead, Joel Charrow, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|October 27, 2010
Therapeutic goals in the treatment of Fabry diseaseAtul Mehta, Michael L West, Guillem Pintos-Morell, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|October 22, 2010
Effects of enzyme replacement therapy in Fabry disease--a comprehensive review of the medical literatureOlivier Lidove, Michael L West, Guillem Pintos-Morell, et al.
Molecular Genetics and Metabolism|November 20, 2012
Juvenile-onset motor neuron disease caused by novel mutations in β-hexosaminidaseTyler Mark Pierson, Paola A Torres, Bei-Jin Zeng, et al.
Blood Cells, Molecules & Diseases|June 22, 2014
A Phase 3, multicenter, open-label, switchover trial to assess the safety and efficacy of taliglucerase alfa, a plant cell-expressed recombinant human glucocerebrosidase, in adult and pediatric patients with Gaucher disease previously treated with imigluceraseGregory M Pastores, Milan Petakov, Pilar Giraldo, et al.
JIMD Reports|September 6, 2021
Abnormal N-glycan fucosylation, galactosylation, and sialylation of IgG in adults with classical galactosemia, influence of dietary galactose intakeEileen P Treacy, Sebastian Vencken, Annet M Bosch, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|February 17, 2005
Individualization of long-term enzyme replacement therapy for Gaucher diseaseHans C Andersson, Joel Charrow, Paige Kaplan, et al.
Pediatrics|January 2, 2009
Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis ILorne A Clarke, J Edmond Wraith, Michael Beck, et al.
Journal of Medical Genetics|February 9, 2012
Fabry International Prognostic Index: a predictive severity score for Anderson-Fabry diseaseDerralynn A Hughes, Mia Malmenäs, Patrick B Deegan, et al.
Molecular Genetics and Metabolism|March 6, 2007
The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type IGregory M Pastores, Pamela Arn, Michael Beck, et al.
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