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AVAIL SAMPLE @ Gaucher's Disease - Pipeline Review, H2 2015
2014 news and press releases related to Gaucher's Disease discussed in this research include:
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.
Surgical management of spinal involvement in children and adolescents with Gaucher's disease.
About one-third of the cases of Gaucher's Disease are diagnosed within the first decade, while the remainder are diagnosed during adulthood.
A review of the Gaucher's Disease products under development by companies and universities/research institutes based on information derived from company and industry-specific sources.
People with Gaucher's disease inherit a defective enzyme that cannot break down a fatty substance called glucocerebroside.
Now, in the largest studies of their kind, two research teams have discovered significant associations between particular combinations of genetic mutations and the severity of Gaucher's disease.
Vevesca(TM) fulfilled the designation criteria as Gaucher's disease is a life threatening and chronically debilitating condition that affects not more than 5 in 10,000 persons within the EU.
Most of the estimated 10,000 to 20,000 people in the United States with Gaucher's disease have relatively mild cases of type 1.
Rather than a new type of GCR, the next major development in the treatment of Gaucher's disease is likely to be gene therapy," Termeer said.
The report provides an in-depth analysis of Gaucher's disease, Fabry disease, Pompe disease, mucopolysaccharidosis VI and Niemann-Pick type C.