Journal article

Interruption of enzyme replacement therapy in Gaucher disease

J Goldblatt, JM Fletcher, J McGill, J Szer, M Wilson

South African Medical Journal | SA MEDICAL ASSOC | Published : 2016

Abstract

In Australia, 58 patients with Gaucher disease were managed by a Gaucher Disease Advisory Committee (GDAC) through a centrally adminis-tered national programme, the Life Savings Drug Program (LSDP). In June 2009, Genzyme Corporation, which manufactures imiglucerase (Cerezyme), the only enzyme replacement therapy (ERT) registered for the treatment of Gaucher disease in Australia at that time, announced that due to a viral contamination problem there would be no further shipments of Cerezyme to Australia prior to the end of 2009. The GDAC allocated available drug supplies in order to maintain treatment to those most in need on a hierarchal clinical severity basis. A cohort of 24 patients with ..

View full abstract

Grants

Funding Acknowledgements

Jack Goldblatt has received travel grants for attendance at conferences, as well as honoraria for presentations at conferences from Shire HGT, Sanofi/Genzyme Corporation, Actelion Pharmaceuticals, Pfizer/Protalix. However, he is not and has never been an employee of any company involved in the management of Gaucher disease, and neither he nor any of his dependents have any proprietary interest in any company producing products used in the management of Gaucher disease. Jeffrey Szer has received travel grants for attendance at conferences, as well as honoraria for presentations at conferences from Shire HGT, Sanofi/Genzyme Corporation, Actelion Pharmaceuticals, Pfizer/Protalix. However, he is not and has never been an employee of any company involved in the management of Gaucher disease, and neither he nor any of his dependents have any proprietary interest in any company producing products used in the management of Gaucher disease. Jim McGill has received travel grants for attendance at conferences, and honoraria from Sanofi/Genzyme Corporation for presentations given at and chairing conferences have been paid to his hospital. However, he is not and has never been an employee of any company involved in the management of Gaucher disease, and neither he nor any of his dependents have any proprietary interest in any company producing products used in the management of Gaucher disease.