Comparative evaluation of α-galactosidase A infusions for treatment of Fabry disease

Robert J. Hopkin, John Bissler, Gregory A. Grabowski

Research output: Contribution to journalReview article

30 Citations (Scopus)

Abstract

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, α-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 direct comparisons difficult. Here, the strengths and weaknesses of these trials are compared: achievement of stated endpoints, safety and, potential efficacy. In addition, the need for additional long-term data is emphasized because this is not attainable in short-term trials for a chronic disease.

Original languageEnglish (US)
Pages (from-to)144-153
Number of pages10
JournalGenetics in Medicine
Volume5
Issue number3
DOIs
StatePublished - May 1 2003
Externally publishedYes

Fingerprint

Galactosidases
Enzyme Therapy
Fabry Disease
Gaucher Disease
Chronic Disease
Outcome Assessment (Health Care)
Safety
Therapeutics
agalsidase beta
agalsidase alfa

All Science Journal Classification (ASJC) codes

  • Genetics(clinical)

Cite this

Comparative evaluation of α-galactosidase A infusions for treatment of Fabry disease. / Hopkin, Robert J.; Bissler, John; Grabowski, Gregory A.

In: Genetics in Medicine, Vol. 5, No. 3, 01.05.2003, p. 144-153.

Research output: Contribution to journalReview article

Hopkin, Robert J. ; Bissler, John ; Grabowski, Gregory A. / Comparative evaluation of α-galactosidase A infusions for treatment of Fabry disease. In: Genetics in Medicine. 2003 ; Vol. 5, No. 3. pp. 144-153.
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