Amicus Therapeutics Engineering Treatment for Rare Conditions

Global biotechnology firm, Amicus Therapeutics focuses on therapies for rare and orphan diseases such as Epidermolysis Bullosa, CDKL5 deficiency, and Lysosomal Storage Disorders. The company targets programs that have three common factors. First, it should be in the rare disease category, have available clinical data that prove significant benefits for patients and incorporates top-notch technology. The premier treatment option from Amicus Therapeutics is the migalastat, a precision medicine for Fabry disease treatment. When administered, migalastat factors in the genetic diagnosis. Another product in the late-stage development is the SD-101, a therapy for treatment of Epidermolysis Bullosa. Visit Market Watch to know more about Amicus Therapeutics.

With the CHART technologies and biologics, Amicus Therapeutics is developing enzyme replacement therapy products for the treatment of Lysosomal Storage Disorders (LSDs), Fabry and Pompe diseases. ATB200/AT221 used together with pharmacological chaperone is the lead product for Pompe illness. Amicus Therapeutics prioritizes personalized and compassionate care for the patients, families, and caregivers. As such, the company incorporates direct and personal relationships with clients in its organizational culture. Each member of the Amicus Therapeutics team has embraced its vision of improving care.

Passionate about their patients, Amicus Therapeutics has embraced an approach that showcases each treatment story in its offices around the globe. Amicus Therapeutics’ intention in doing so is to encourage other patients and to highlight the possibilities in the treatment of rare diseases. Amicus Therapeutics makes its decisions from the perspective of the clients and the affected friends and family. Each decision addresses the benefit to the patient and the likely risks. Amicus Therapeutics values ethics as much as it values its patients. The company upholds high standards of integrity and complies with the regulations in the sector.

In 2007, Amicus Therapeutics became a publicly traded company. Before Radius Ventures, New Enterprises Associations and Canaan Partners financed the company. The company received recognition for having the most extensive portfolio of pharmacological chaperones in the industry in 2014. Amicus Therapeutics has collaborated with major pharmaceutical companies like JCR Pharmaceuticals and GlaxoSmithKline in research and development of treatment options.

With offices in Germany, Spain, France, Italy, the Netherlands and the US, Amicus Therapeutics is looking to transform the treatment of rare conditions. Know more about Amicus Therapeutics on

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