Meeting Resources

Learn more about the meeting

View the Meeting Agenda

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Special Thanks

We would like to thank our collaborating organizations for this important effort, which include the Dana’s Angels Research Trust, Firefly Fund, Hide & Seek Foundation, Hope for Marian, Johnathan’s Dream, National Niemann-Pick Disease Foundation, and Niemann-Pick Canada.

We would also like to thank the organizations that are assisting in underwriting the cost of the PFDD meeting, which include Mallinckrodt Pharmaceuticals, Orphazyme, StrideBio, Inc., and the Together Strong NPC Foundation. These organizations will not receive any special access or benefits in regard to the meeting or related materials, and the PFDD meeting and access to the Voice of the Patient report are open to all other interested industry, foundation, and government stakeholders.