Dear Guest:

At Amicus, we are committed to supporting members of the genetic diseases communities by searching for new and better therapies, initiating clinical trials for those therapies as our research advances, and providing useful information and resources to help manage the daily challenges of living with a rare genetic disease. Patient Advocacy works in partnership with patient organizations and individuals and families with genetic diseases to fulfill these commitments. Through building strong alliances, excellent communication and collaboration within the rare genetic diseases communities, Patient Advocacy hopes to ease the difficulties encountered by members in accessing support and services across their disease experience and in meeting their on-going need for improved therapies.

In the Patient Advocacy section of the Amicus Therapeutics’ web site, you will find valuable information about the genetic diseases in which we are actively developing an investigational drug program using the pharmacological chaperone technology. Within each genetic disease page we provide information about (1) the disease, (2) our investigational drug and its status in clinical research, (3) external resources and services that are available to support the disease community, and (4) selected events that are happening within the genetic disease communities to educate, support, and advocate on behalf of its members. As we continue to move forward in development, we will continue to add services aimed at meeting the needs of the rare genetics disease community. We invite you to offer comments and suggestions.

Please feel free to contact us with questions and comments as you navigate through your experience with the rare genetic diseases communities, Amicus, and/or pharmacological chaperone technology: patientadvocacy@amicustherapeutics.com.