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Emery and Rimoin's Principles and Practice of Medical Genetics and Genomics by Pyeritz Reed E.;Korf Bruce R.;Grody Wayne W.; & Bruce R. Korf & Wayne W. Grody

Author:Pyeritz, Reed E.;Korf, Bruce R.;Grody, Wayne W.; & Bruce R. Korf & Wayne W. Grody , Date: March 5, 2020 ,Views: 172

Emery and Rimoin's Principles and Practice of Medical Genetics and Genomics by Pyeritz Reed E.;Korf Bruce R.;Grody Wayne W.; & Bruce R. Korf & Wayne W. Grody

Author:Pyeritz, Reed E.;Korf, Bruce R.;Grody, Wayne W.; & Bruce R. Korf & Wayne W. Grody
Language: eng
Format: epub
ISBN: 9780128126844
Publisher: Elsevier Ltd.
Published: 2018-11-21T00:32:43+00:00


Table 7.1

ERT Lessons Learned From Gaucher DiseaseProof of Concept: ERT Works

Safe and generally well tolerated

Minimal immunologic complications (cross-reactive immunologic material positive (CRIM+) patients)

Dose-dependent enzyme delivery

Mannose receptor-mediated enzyme uptake

Inability to cross blood–brain barrier

Dose-dependent substrate clearance

Unexpected reversal of lysosomal storage in organs

Rapid reaccumulation off therapy: lifetime therapy

Need to treat early to prevent irreversible disease



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Emery and Rimoin's Principles and Practice of Medical Genetics and Genomics by Pyeritz Reed E.;Korf Bruce R.;Grody Wayne W.; & Bruce R. Korf & Wayne W. Grody.epub


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