Why Amicus Therapeutics Maybe the Next Solution for Rare Diseases

When it comes to biotechnology, Amicus Therapeutics has earned its place globally to become the leader in offering therapies focusing on rare as well as orphan diseases. Based in Cranbury, the company’s has a development pipeline that efficiently treats various human genetic diseases through its range of products. One of its lead products, migalastat, is one of the medicines that treat genetic disorders of the Fabry disease. Amicus Therapeutics leverages on biologics as well as Chaperone-Advances Replacement Therapy to offer treatment for other genetic diseases such as Lysosomal Storage Disorders (



Despite its reliance on other manufacturers for its products, the company continues to provide innovative technology platform in developing solutions for rare diseases. The treatments at Amicus Therapeutics are made possible through targeting of mutated proteins which offer healing that is beyond the mere diseases. One of the patients who has been through the company, Alex, who is 47 years attests to the effective treatment offered by Amicus Therapeutics. His condition was five years long quest after diagnosis with Fabry disease which scared not only him but also his family. Following his enrollment into a clinical study at Amicus Therapeutics in 2006, the family is now optimistic that even his daughter will be on the route to recovery.



The company goes beyond offering treatment for patients to collaborating with other organizations, caregivers, health professionals as well as individual patients to ensure combined effort in giving the best treatment for their patients. Amicus’ dedication is to develop other treatments from this advocacy which goes a long way in providing better therapies. Further, the company hopes to continually improve the provision of useful information as well as resources meant to help patients manage the challenges that come along with the diagnosis of rare, genetic diseases (YahooFinance). Through advocacy, the company also hopes to bring patients on board to be part of designing clinical trials for therapies with the advancement of research during the process of drugs development. The role of Amicus Therapeutics can, therefore, be termed as critical in helping treat genetic diseases.

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