Amicus Therapeutics Is The Champion That Battles Rare Diseases

There is a population of people of less than 200,000 that suffer from rare and orphan diseases. These maladies can affect the families as much as it affects the patient. Amicus Therapeutics has discovered the means for managing and living with rare diseases.


The Amicus Therapeutics company has developed specific treatments and programs for patients that suffer from Fabry, Pompe, and Epidermolysis Bullosa. These diseases have clinical studies that have been completed and the research continues.


The Amicus Therapeutics research is working on building pharmacological chaperones for Lysosomal Storage Disorders or LSD’s. The company’s’ scientific research has led to Enzyme Replacement Therapy or ERT combined with other medications to enhance the treatment of rare and orphan diseases.


The Amicus Therapeutics company provides information for resources, services and support for the patient and their family focused on how to live and manage their disease. The mutated proteins have become the targets to design and develop treatments for the genetic diseases of Lysosomal Storage Disorders or LSD’s. Through their exhaustive research, they are certain they will find a cure.


The Callidus Biopharma is now owned and operated by the Amicus Therapeutics Company since November 2013 (SeekingAlpha). The Enzyme Replacement Therapy treatments and Creative Property for the Pompe Disease were part of the package. The Scioderm company and stocks were purchased in September 2015 for $947 million cash.


Support For The Dynamic Duo’s Of Research


* The Alzheimer’s Drug Discovery Foundation gave $210,300 to Amicus Therapeutics to support their pre-clinical research. The Icahn School of Medicine at Mount Sinai through the Alzheimer’s Disease Research Center will be working together on the projects.

* The Michael J. Fox Foundation is an advocate for the research of the Amicus Therapeutics company and gave them $500,000. The David Geffen School of Medicine at UCLA and Amicus Therapeutics research teams will conduct the research together.

For more about Amicus Therapeutics visit

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