References in periodicals archive ?
Indeed, life may be tough for individuals who suffer from Gaucher's disease, a rare and inherited disorder wherein the deficiency of enzyme called beta-glucocerebrosidase (that is produced in our cells) results in the accumulation of a fatty substance called glucocerebroside into a sugar and a simpler fat molecule throughout the body, more often in the bone marrow, spleen and liver.
* The report reviews key players involved in the therapeutics development for Gaucher's Disease and enlists all their major and minor projects
* Identify and understand important and diverse types of therapeutics under development for Gaucher's Disease.
"This study is likely to motivate clinical trials for the treatment of neuropathic lysosomal storage diseases, including Gaucher's disease, where the current standard of care, enzyme replacement therapy, is ineffective," Nature quoted team leader Dr.
Taliglucerase alfa is currently being provided to Gaucher's patients in the US under an expanded access protocol, as well as to patients in the European Union under a compassionate use protocol, Protalix said.
The drug produces the enzyme--a type of protein that triggers a biochemical reaction-that's deficient in Gaucher's patients.
Shire Pharmaceuticals PLC is also seeking approval in the United States to sell a drug for Gaucher's disease.
Replacement therapy for inherited enzyme deficiency: macrophage-targeted glucocerebrosidase for Gaucher's disease.
Upper limb involvement in patients with Gaucher's disease.
The recent arrival on the market of a modified glucocerebrosidase (Ceredase) for Gaucher's Disease and last year's approval of PEG-adenosine deaminase for severe combined immunodeficiency disease illustrate the growing capabilities of the pharmaceutical industry to provide endogenous compounds that patients are lacking.
Brady first proposed enzyme-replacement therapy for Gaucher's disease 25 years ago.