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Mar24
Preclinical Data of Amicus Therapeutics’ Plicera™, Promising against Gaucher Disease

Preclinical studies of Amicus Therapeutics (706165)’ Plicera™ (isofagomine tartrate, AT2101) revealed that it can increase levels of the target enzyme in cells derived from a patient with the N370S mutation and in mice that express the L444P mutation – the two most common mutations associated with Gaucher's disease.

Amicus.gifThe most commonly diagnosed lysosomal storage disorder - Gaucher's disease - is caused by inherited genetic mutations in the GBA gene, resulting to deficient activity of the enzyme acid beta-glucosidase or glucocerebrosidase (GCase).

Plicera™ is designed to selectively bind to and stabilize GCase.

The complete clinical data have been presented at the American College of Medical Genetics (ACMG) annual meeting (March 21-25, Nashville, TN).

Read the full press release.


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« Promising Research Data Suggest Potential of ImmuneRegen BioSciences’ Homspera™ as Co-therapeutic Agent | Main | CTI Filed USFDA SPA for Phase III Trial of XYOTAX™ in Women Lung Cancer »

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