Gaucher's disease


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Words related to Gaucher's disease

a rare chronic disorder of lipid metabolism of genetic origin

References in periodicals archive ?
Because of the progress and great strides that have been made, the organization stressed that Filipino children may never have to suffer the debilitating and painful affects of Gaucher's disease.
This report provides comprehensive information on the therapeutic development for Gaucher's Disease, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases.
Gynecologic and obstetric aspects of Gaucher's disease: a survey of 53 patients.
Our study revealed that of 33 cases of inborn errors of metabolism, 23 cases (70%) were reported as glycogen storage disorders, 5 (15%) cases as Gaucher's disease and 5 cases (15%) Niemann-Pick's disease.
* Coverage of the Gaucher's Disease pipeline on the basis of route of administration and molecule type.
Pulmonary hypertension in type 1 Gaucher's disease: genetic and epigenetic determinants of phenotype and response to therapy.
London, May 10 (ANI): Scientists at The Scripps Research Institute have shed light on a mechanism that enables a potential treatment for Gaucher's disease and other lysosomal storage diseases.
<![CDATA[ An Israeli firm has passed the first hurdle in gaining FDA approval for a new treatment for Gaucher's disease.]]>
The deal also gives Pfizer worldwide rights, except in Israel, to sell a genetically engineered drug called taliglucerase alfa to treat a rare inherited condition known as Gaucher's disease. Caused by deficiency of a particular enzyme, it damages the liver and bone marrow, sometimes leading to death.
has licensed global rights to a treatment for Gaucher's disease from Protalix.
Protalix expects to complete the company's Phase III trial of prGCD for the treatment of Gaucher's disease in September, to report top-line results in October and to complete the NDA filing before the end of the year.
Replacement therapy for inherited enzyme deficiency: macrophage-targeted glucocerebrosidase for Gaucher's disease. N Engl J Med 1991;324:1464-70.