Amicus Therapeutics in the Cure of Rare and Orphan Diseases

Amicus Therapeutics is a public American biopharmaceutical company founded on February 4th, 2002. The company has its headquarters in Cranbury, New Jersey. Amicus Therapeutics is a global biotechnology company at the forefront of improved therapies to treat a variety of desolating rare and orphan diseases (http://weeklyopinion.com/2017/08/amicus-therapeutics-announces-the-launch-of-new-drug/). This kind of diseases includes; Epidermolysis Bullosa (EB,) Pompe diseases as well as Lysosomal Storage Disorders and the Fabry disease.

 

The amicus therapeutics company has an exclusive set of programs in technologies and medicines in development for people living with these kinds of rare orphan diseases. The company has developed a platform Chaperone-Advanced Replacement Therapy that aims at enzyme replacement therapy (ETR) products for Fabry disease, Pompe disease, and other lysosomal storage disorders. The company is also working to develop a biologics program ATB200/AT2221 which will uniquely be administered in the treatment of Pompe disease.

 

Amicus Therapeutics Company uses advanced technology, and it has seen it at the forefront of developing therapies in addressing and meeting the needs of the rare and orphan diseases (SeekingAlpha). Apart from developing a pipeline towards the treatment of these rare diseases, amicus professionals act as a bridge between the patients, their families and the resources also offer access to support and services across their disease experience.

 

The amicus therapeutics finally has its goal to make a meaningful difference in the lives of these patients. It has greatly contributed to the development of the first Fabry disease medicine Galafold through EMA and is working to obtain approval for further studies. Also, it is expecting a pivotal three phrase trial readouts in epidermolysis bullosa (ER).

More about Amicus Therapeutics on Crunchbase and Facebook

 

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