The rare diseases relief through Amicus Therapeutics

Therapeutics is an American company dealing with biopharmaceuticals. It is located in Cranbury, New Jersey. The company was funded by various venture capital firms like Radius Ventures and Canaan Partners. Their main concentration is on occasional and orphan diseases especially the lysosomal storage disorders. Their product development is totally dependent on Chaperone Advanced Replacement Therapy. The company was noted to have the broadest collection of small molecule pharmacological chaperones in the pharmaceutical business. It collaborate with GlaxoSmithKline and JCR Pharmaceuticals for three years to investigate coformulation with recombinant alpha-galactosidase. Amicus Company has no manufacturing capability of its own rather it relies on the contract manufacturing.


The company is focused on the rare opportunity of achieving more through seeking to make a difference in the lives of patients. The biotechnology company has a vigorous expansion pipeline of handlings for a wide assortment of people genetic diseases. Patients with Fabry disease should be relieved by their leading product, migalastat. The drug is personalized to assist late stage patients suffering from Fabry. Recent updates state that Amicus Therapeutics have been confirmed to submit a new drug application for the oral precision medicine migalastat for Fabry disease. The willingness of the FDA to review migalastat data depicts patient-centric therapeutic development ( The break through is appreciated for the employees that have worked tirelessly committed to finding relief for the Fabry disease patients.


Amicus Therapeutics participated in the MDA 2017 scientific conference in which they presented preliminary clinical data in Pompe disease. The conference concentrated on finding better understanding diseases causes, identifying new therapeutics targets and even discussing new advances in the preclinical and clinical research. All of these efforts are channeled towards finding the treatment for neuromuscular diseases. Their leading program is ATB200/AT2221 which is a exclusively concocted pompe illness ERT which will be provided together pharmacological chaperone. The other programs include development pipeline, Fabry disease and Epidermolysis Bullosis. The Chief Financial Officer Mr. Chip Baird in his speech expressed his honor to maximize the financing needed to support therapies since they could bring major changes to patients and families being affected by these rare diseases.

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