[Response criteria for enzyme substitution in Gaucher disease].

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Article

[Response criteria for enzyme substitution in Gaucher disease].

Monatsschrift Kinderheilkunde : Organ der Deutschen Gesellschaft fur Kinderheilkunde - 1 Oct 1992

Berthold F, Sieverts H, Benz-Bohm G, Landwehr P, Harzer K

Abstract excerpt

Recently the intravenous enzyme replacement therapy with modified beta-glucocerebrosidase has become available for patients with M. Gaucher. We report here the considerable improvement of activity and vigor in a 5 year old girl with type 1 M. Gaucher administering 60 IU/kg every two weeks for 6 m...

Topics

Acid Phosphatase
Alleles
Bone Marrow
Child, Preschool
Female
Gaucher Disease
Genotype
Glucosylceramidase
Humans
Infusions, Intravenous
Magnetic Resonance Imaging
Mutation
Peptidyl-Dipeptidase A

Source

Open in PubMed

Identifiers

PMID: 1331780

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 30 Aug 2026. Prepared record publication-1331780-2026-08-30.1. Manifest publication-graph-cohort-2026-08-30.1.

Source receipt: PubMed via Helena Literature Corpus, retrieved 30 Aug 2026.

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