Display Settings:

Format

Send to:

Choose Destination
See comment in PubMed Commons below
J Inherit Metab Dis. 2001;24 Suppl 2:89-96; discussion 87-8.

Lessons learned from the development of enzyme therapy for Gaucher disease.

Author information

  • 1Department of Human Genetics, University of Pittsburgh, Pennsylvania 15261, USA. jbarrang@helix.hgen.pitt.edu

Abstract

Enzyme replacement therapy for the lysosomal storage disorders derives its impetus from the successes achieved in the treatment of Gaucher disease. After nearly two decades of persistent but unsuccessful efforts, the promise of therapy through enzyme replacement was losing credibility. Then, the fortunate intersection of two different lines of scientific research produced the necessary breakthrough. The dramatic responses to enzyme replacement therapy in patients with Gaucher disease made it immediately clear that this treatment approach was a success. Furthermore, the large number of patients with the disorder guaranteed commercial involvement. The lessons learned from the development of enzyme replacement therapy for Gaucher disease are broadly applicable to other lysosomal storage diseases and will be reviewed in this paper.

PMID:
11758684
[PubMed - indexed for MEDLINE]
PubMed Commons home

PubMed Commons

0 comments
How to join PubMed Commons

    Supplemental Content

    Loading ...
    Write to the Help Desk