Amicus Therapeutics; Passion and Progress in Combating Rare Disease

Amicus Therapeutics is dedicated to staying in the vanguard of modern biotechnology-based therapy. Since 2002, we have continuously pushed forward in the development of cutting-edge treatment to combat a variety of disorders. Our primary focus has been on rare and orphan diseases, with particular attention given to Lysosomal Storage Disorders, Epidermolysis Bullosa, and CDKL5 Deficiency. Utilizing Chaperone-Advanced Replacement Therapy, (CHARTtm), to develop a host of enzyme replacement therapies, Amicus Therapeutics has positioned itself as a leader in the world of small molecule pharmacological chaperones.Lysosomal Storage Disorders, Epidermolysis Bullosa, and CDKL5 Deficiency


Through a great deal of hard work, research and development, Amicus Therapeutics has grown and evolved to become a leading innovator in the fight against rare disease (YourBeautyCraze). Widely recognized as holding one of the broadest portfolios of small molecule pharmacological chaperones in the industry, our team strives to support the rare disease community on multiple fronts. Our approach to patient advocacy includes a four-fold approach to healing that includes:


Direct and sustained contact with patients and their families, such that two way communication is established for the benefit of all involved. We take the time to learn from our patients and their families and we help them.


-Constant research to improve our current therapies, and to create new ones.


-Soliciting and utilizing patient input during development and trial processes of out therapies.


-Providing up to date resources for all our patients and their families, which help them to navigate the day to day challenges of combating a rare disease.


Amicus Therapeutics also actively seeks out opportunities to help educate the broader community about the realities of rare diseases. In doing this, we are able to create a more comprehensive support network to benefit those who are currently combating a rare disease. We believe that healing is something that must happen on multiple levels, which requires efforts above and beyond simply treating the symptoms of an illness.


We understand the challenges and the difficulties of coping with a rare disease. There is no single checklist that every patient may follow to recover, or maintain their quality of life. Amicus Therapeutics sees every patient as an individual with unique needs. Our mission, and our passion, is to meet these needs for everyone who must cope with the challenges of a rare disease.

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