Amicus Therapeutics, Inc., a biotechnology company, focuses on discovering, developing, and delivering novel medicines for rare diseases in the United States and internationally. The company’s ...

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Amicus Therapeutics’ first oral drug for the rare condition Fabry disease has been approved by the FDA, giving patients an alternative to conventional injected enzyme replacement therapy.

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However, enthusiasm is tempered by discussions about key barriers. Experts at ACR 2024 noted that the requirement for apheresis to collect patient T-cells poses logistical and clinical challenges.