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Deborah Elstein, Aya Abrahamov, Anat Oz, Naama Arbel, Hagit Baris, Ari Zimran
BACKGROUND: Lifelong intravenous (IV) enzyme replacement therapy (ERT) every other week for Gaucher disease is appreciated as decreasing quality of life in a palpable way. OBJECTIVE: To review the Israeli experience with the home therapy option for IV velaglucerase alfa (Shire, Lexington MA USA) infusions every-other-week in the clinical trial context, in the early access program (EAP) during a shortage with the standard commercial ERT, and currently with the commercially available drug (VPRIV, Shire)...
December 2015: Blood Cells, Molecules & Diseases
Derralynn A Hughes, Derlis E Gonzalez, Elena A Lukina, Atul Mehta, Madhulika Kabra, Deborah Elstein, Isaac Kisinovsky, Pilar Giraldo, Ashish Bavdekar, Thomas N Hangartner, Nan Wang, Eric Crombez, Ari Zimran
Type 1 Gaucher disease is an inherited lysosomal enzyme deficiency with variable age of symptom onset. Common presenting signs include thrombocytopenia, anemia, hepatosplenomegaly, bone abnormalities, and, additionally in children, growth failure. Fifty-seven patients aged 3-62 years at the baseline of two phase III trials for velaglucerase alfa treatment were enrolled in the single extension study. In the extension, they received every-other-week velaglucerase alfa intravenous infusions for 1.2-4.8 years at 60 U/kg, although 10 patients experienced dose reduction...
July 2015: American Journal of Hematology
Hongping Ye, John Hill, Ashley C Gucinski, Michael T Boyne, Lucinda F Buhse
Gaucher disease, the most common lysosomal metabolic disorder, can be treated with enzyme replacement therapy (ERT). Recombinant human glucocerebrosidase imiglucerase (Cerezyme(®)), produced in Chinese hamster ovary cells, has been used for ERT of Gaucher disease for 20 years. Another recombinant glucocerebrosidase velaglucerase alfa (VPRIV), expressed in a human fibroblast cell line, was approved by the US Food and Drug Administration in 2010. The amino acid sequence difference at residue 495 of these two products is well documented...
March 2015: AAPS Journal
Deborah Elstein, Derralynn Hughes, Ozlem Goker-Alpan, Miriam Stivel, Hagit N Baris, Ian J Cohen, Sorina Granovsky-Grisaru, Arnon Samueloff, Atul Mehta, Ari Zimran
AIM: Pregnancy and delivery are affected by and - in turn - impact signs and symptoms of Gaucher disease (GD). Prior to enzyme replacement therapy (ERT), the reported missed abortions rate was 25%, with worsening of anemia and thrombocytopenia, with increased frequency of post-partum hemorrhage, puerperal fever and bone crises during pregnancy. ERT with imiglucerase reduced these adverse events. Velaglucerase alfa (VPRIV), an ERT approved commercially in February 2010, had undergone preclinical reproductive toxicity testing and proven to be safe and effective in phase I/II and III clinical trials...
April 2014: Journal of Obstetrics and Gynaecology Research
Ritesh Thekkedath, Alexander Koshkaryev, Vladimir P Torchilin
AIM: We hypothesized that liposomes modified with lysosomotropic octadecyl-rhodamine B (Rh) and loaded with therapeutic glucocerebroside velaglucerase alfa (VPRIV™) will improve lysosomal delivery of the enzyme into Gaucher's cells. MATERIALS & METHODS: Confocal microscopy and flow cytometry were used to evaluate the ability of Rh-modified liposomes loaded with VPRIV to improve the lysosomal targeting in monocyte-derived macrophages and Gaucher's fibroblasts. RESULTS: Confocal microscopy demonstrated that Rh-modified liposomes localized primarily in the lysosomes...
July 2013: Nanomedicine
Deborah Elstein
Gaucher disease is inherited as an autosomal recessive disorder. The absence of β-glucocerebrosidase whose purpose is to cleave the glucose from ceramide results in accumulation of glucocerebroside; storage of this glycolipid results in Gaucher disease. There is tremendous clinical heterogeneity: prediction of onset of symptoms (if at all), which organs will be affected, and the degree of severity of the signs and symptoms are areas of current research. Lysosomal storage diseases may be treatable by enzyme replacement therapy...
June 2011: Current Pharmaceutical Biotechnology
Timothy M Cox
Gaucher disease is a rare inborn error of glycosphingolipid metabolism due to deficiency of lysosomal acid β-glucocerebrosidase; the condition has totemic significance for the development of orphan drugs. A designer therapy, which harnesses the mannose receptor to complement the functional defect in macrophages, ameliorates the principal clinical manifestations in hematopoietic bone marrow and viscera. While several aspects of Gaucher disease (particularly those affecting the skeleton and brain) are refractory to treatment, enzyme (replacement) therapy has become a pharmaceutical blockbuster...
2010: Biologics: Targets & Therapy
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May 3, 2010: Medical Letter on Drugs and Therapeutics
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May 3, 2010: Medical Letter on Drugs and Therapeutics
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