How we manage Gaucher Disease in the era of choices
Shoshana Revel-Vilk, Jeff Szer, Atul Mehta, Ari Zimran
BRITISH JOURNAL OF HAEMATOLOGY | WILEY | Published : 2018
Treatment of Gaucher Disease (GD) is now beset with the abundance of therapeutic options for an individual patient, making the choice of therapy complex for both expert and non-expert clinicians. The pathogenesis of all disease manifestations is a gene mutation-driven deficiency of glucocerebrosidase, but the clinical expression and response of each of the clinical manifestations to different therapies can be difficult to predict. Enzyme replacement therapy has been available since 1991 and is well-established, with known efficacy and minimal toxicity. Of interest, the three available enzymes are distinct molecules and were registered as new products, not biosimilars. Oral substrate reductio..View full abstract
AZ has received honoraria and speaker fees from Shire, Pfizer and Sanofi/Genzyme. SRV has received honoraria, speaker and travel fees from Shire, Pfizer and Sanofi/Genzyme. The SZMC Gaucher Clinic receives support from Sanofi/Genzyme for participation in the ICGG Registry, from Shire for the GOS Registry and from Pfizer for TALIAS. The Clinic also receives research grants from Shire and from Pfizer. AM has received research support, honoraria and speaker fees from Shire, Pfizer and Sanofi/Genzyme. JS has received honoraria and speaker fees from Shire, Pfizer and Sanofi/Genzyme.