FOLD - Amicus Therapeutics Inc.

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Amicus Therapeutics, Inc., headquartered in Philadelphia, Pennsylvania, is a biotechnology firm dedicated to the discovery, development, and delivery of treatments for rare diseases. Established in 2002, the company focuses on addressing unmet medical needs through innovative therapies.

The cornerstone of Amicus Therapeutics' portfolio is Galafold, an oral precision medicine approved for adults diagnosed with Fabry disease who possess specific genetic mutations. This therapy exemplifies the company's commitment to leveraging genetic insights to tailor treatments for individual patients.

In addition to Galafold, the company is advancing AT-GAA, a promising treatment approach for Pompe disease, a rare genetic disorder affecting muscle function. Amicus Therapeutics is also exploring enzyme replacement therapies and gene therapies targeting various rare diseases, including CLN3 and CDKL5, through clinical trials aimed at evaluating safety and efficacy.

Collaboration and licensing agreements with esteemed institutions like Nationwide Children's Hospital, University of Pennsylvania, and GlaxoSmithKline bolster Amicus Therapeutics' research and development efforts. These partnerships underscore the company's strategic approach to harnessing scientific expertise and advancing novel therapies to improve the lives of patients affected by rare diseases worldwide.

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