Category Archives: Fabry

Allow Me To Re-Introduce Amicus Therapeutics

Published / by healthadmin

Amicus Therapeutics is a world wide biotechnology company based in Cranbury, New Jersey that was founded on February 4, 2002. It is on the cutting edge of advanced therapies and strategies for treating a wide array of annihilative rare and orphan diseases.

 

What particular diseases does Amicus Therapeutics focus on?

Amicus Therapeutics takes aim at a group of disorders that are collectively identified as lysosomal storage diseases (LSD).

 

What exactly are lysosomal storage diseases (LSD)?

An array of approximately 50 unique metabolic disorders that are inherited and are a result of imperfections found within the lysosomal function. Sacs of enzymes called lysosomes exists within cells that digest large molecules and then transfer the remnants on to other sections of the cell for reuse. The procedure needs several important enzymes and if any of these enzymes are defective due to mutation the larger molecules gather inside of the cell and eventually eradicate it.

 

What is CHART® and how does it work?

Chaperone-Advanced Replacement Therapy is Amicus Therapeutics’ main produce aimed at the development of enzyme replacement therapies.

 

Amicus Therapeutics VS Fabry

Amicus Therapeutics most promising candidate for the fight against Fabry is their product migalastat. It is a pharmacological chaperone treatment that targets the stabilization of endogenous mutant alpha-galactosidase along with the progression of migalastat monotherapy. They formed up in conjunction with JCR and Glaxo-SmithKline for three years (2010 to 2013) as the team analyzed the co-formulation with recombinant alpha-galactosidase (JR-051).

 

Expansion of Amicus Therapeutics

In 2008 Amicus Therapeutics expanded from one site located in New Jersey to open another facility in San Diego (http://www.marketwatch.com/investing/stock/fold).

In 2010 The Michael J. Fox Foundation blessed Amicus Therapeutics with a $500,000 grant so they could continue their studies in conjunction with the David Geffen School of Medicine at UCLA. In the same year, they received $210,300 grant that came from the Alzheimer’s Drug Discovery Foundation and the Alzheimer’s Disease Research Center at Mount Sinai.

 

Acquiring Competitors

2013 Callidus Biopharma

2015 Scioderm

 

Definitely, a company to keep your eye on Amicus Therapeutics offers a broad spectrum of treatment options for those suffering from human genetic diseases (Yahoo).