Emerging Treatments and Future Outcomes

Burrow, T.; Grabowski, Gregory A.

doi:10.1002/9781118514672.ch22

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2015

2024

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Cited by 3 publications

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“…Predominantly forms of enzyme replacement are in use, such as allogeneic haematogenous stem cell transplant (HSCT) or infusions of recombinant humanized enzyme replacement therapies (rhERT) [4,5]. Therapies in development include gene therapies and small molecule therapies such as chaperone molecules [6,7].…”

Section: Introductionmentioning

confidence: 99%

Lysosomal Enzyme activity: Establishing Reference Intervals from Patient Data

Mordaunt,

Stark,

Fietz

et al. 2024

Preprint

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Objectives: To develop reference intervals for leukocyte lysosomal enzymology. Design: Standards-based establishment of reference intervals by analysis of 7851 for 17 assays of 15 lysosomal enzymes in leukocytes and plasma (January 1985 through September 2013). Methods: Reference intervals were calculated using MedCalc for Windows version 12.5 (MedCalc Software, Ostend, Belgium), according to the Clinical and Laboratory Standards Institute C-28A protocol (CSLI-C28A). Results and Conclusion: Evidence-based reference intervals are presented in tabular form for seventeen assays of fifteen lysosomal enzymes.

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