Patient Support

We’re committed to a compassionate and systematic approach to engaging with and supporting patients. We start by listening to and learning from patient communities to help form a clear understanding of their needs and how we can collaborate on improving the experience of living with serious medical conditions.

博彩平台网址大全 Rare Connections

Rare conditions often come with uncommon challenges, and we’re here to help. 博彩平台网址大全 RareConnections™ was created to help navigate the difficulties of managing serious and rare genetic conditions through a wide array of product support services throughout your treatment journey.

Our dedicated and experienced RareConnections team members will provide guidance on how you can gain access to 博彩平台网址大全 medicines by:

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Patient Community Engagement

We know that helping to improve the lives of people living with genetic conditions takes more than scientific innovation alone – it also requires close collaboration with patient and advocacy communities.

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RARE Scholars Program

27 Scholarships Awarded since 2018

Our RARE Scholars program provides financial support to U.S. college students living with one of several rare genetic conditions – achondroplasia, Batten disease, hemophilia A, mucopolysaccharidosis (MPS) or phenylketonuria (PKU). Since its inception in 2018, the program has awarded more than $100,000 to 22 students who have demonstrated exceptional leadership and engagement in school and community activities.

Applications are now open for the 2024-25 academic year.

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Trees of Life

When a patient was inspired by the Pont des Arts in Paris, we were inspired to create the Trees of Life, an art installation that symbolizes our steadfast commitment to the rare disease community. Branches are filled with locks dedicated to those impacted by rare diseases as well as those who have made a difference in the development and advancement of therapies that treat genetic conditions.