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In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
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[Current status and future prospect of enzyme replacement therapy for Fabry disease]

Toya Ohashi1,2

  • 1Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School for Medicine.

Rinsho Shinkeigaku = Clinical Neurology
|May 31, 2019
PubMed

Keywords:
Fabry diseaseagalsidase alphaagalsidasse betaenzyme replacement therapypharmacological chaperon

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