Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Comparative evaluation of alpha-galactosidase A infusions for treatment of Fabry disease.

Genetics in medicine : official journal of the American College of Medical Genetics - 1 Jan 2000

Hopkin Robert J, Bissler John, Grabowski Gregory A

Abstract excerpt

Enzyme therapy has proven safe and effective in preventing and reversing many manifestations in patients with Gaucher disease. On the basis of this success, enzyme therapy is now becoming a reality for Fabry disease, alpha-galactosidase A deficiency. Two products, agalsidase alpha and beta, have been tested in pivotal trials. The substantial differences between the study structures and outcome measures have made...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to alpha-Galactosidase
Share to Clinical Trials as Topic

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Drug Evaluation, Preclinical

Share to Fabry Disease

Share to Founder Effect

Share to Isoenzymes

Share to Kidney

Share to Mutation

Source

Identifiers

PMID: 12792421
DOI: 10.1097/01.GIM.0000069509.57929.CD

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 9 closest

Comparative evaluation of alpha-galactosidase A infusions for treatment of Fabry disease.PMID 12792421Read ↗

Enzyme replacement therapy for Fabry disease: lessons from two alpha-galactosidase A orphan products and one FDA approval.Semantic proximity Read ↗

Long Term Treatment with Enzyme Replacement Therapy in Patients with Fabry DiseaseSemantic proximity Read ↗

A 4-year study of the efficacy and tolerability of enzyme replacement therapy with agalsidase alfa in 36 women with Fabry disease.Semantic proximity Read ↗

Enzyme therapy for Gaucher disease: the first 5 yearsSemantic proximity Read ↗

Targeted approaches to induce immune tolerance for Pompe disease therapySemantic proximity Read ↗

Carboxyl-terminal truncations alter the activity of the human α-galactosidase A.Semantic proximity Read ↗

Enzyme replacement therapy in heterozygous females with Fabry disease: results of a phase IIIB study.Semantic proximity Read ↗

Monitoring the clinical and biochemical response to enzyme replacement therapy in three children with Fabry disease.Semantic proximity Read ↗

Synergy between the pharmacological chaperone 1-deoxygalactonojirimycin and the human recombinant alpha-galactosidase A in cultured fibroblasts from patients with Fabry disease.Semantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.