Authors:

  • Ara Parseghian Medical Research Fund at Notre Dame: Sean Kassen, Director; Cindy Parseghian, Founder & President
  • National Niemann-Pick Disease Foundation, Joslyn Crowe, Director
  • Hide & Seek Foundation: Jonathon Jacoby, President
  • Dana’s Angels Research Trust: Phil Marella, Founder & Trustee
  • Hope for Marian, Sara McGlocklin, Founder
  • Niemann-Pick Canada, Cheryl Marcogliese, Vice Chair
  • Johnathon’s Dreams, Rebecca Spencer White, Founder

Consulting Partners:

  • Faegre Baker Daniels: Dave Zook, Lauren Bloch
  • Your Encore: Tim Franson

Clinical Advisors:

  • Elizabeth Berry-Kravis, MD PhD
  • Denny Porter, MD PhD
  • Marc Patterson, MD

Disclosures: Dr. Marc Patterson has received consulting fees and /or fees for participation in data safety monitoring boards from Actelion, Amicus, Cerecor, IntraBio, Novartis Orphazyme, Shire-Takeda, and Vtesse. He owns stock in IntraBio. MCP receives royalties from Elsevier and Wolters Kluwer (Up-To-Date) as an author and editor, respectively, an honorarium and travel expenses from the SSIEM as a journal editor, and a stipend as Editor-in-Chief of the Journal of Child Neurology and Child Neurology Open. He has received, or will receive, grant support from the National Institutes of Health, The National MS society, Actelion, Amicus, Idorsia, Glycomine, Orphazyme, and Shire-Takeda. He has also received philanthropic support from the Peggy Furth Fund at Mayo Clinic for research on lysosomal diseases. Dr. Elizabeth Berry-Kravis has received funding from Seaside Therapeutics, Novartis, Roche, Alcobra, Neuren, Cydan, Fulcrum, GW, Neurotrope, Marinus, Zynerba, BioMarin, Ovid, Retrophin, AMO, Yamo, Acadia, Avexis, Ionis, Ultragenyx, Lumos, GeneTx, and Vtesse/Sucampo/Mallinkcrodt Pharmaceuticals to consult on trial design or development strategies and/or conduct clinical trials in FXS or other NDDs or neurodegenerative disorders, and from Asuragen Inc to develop testing standards for FMR1 testing. All funding to Dr. Kravis. is directed to Rush University Medical Center to support rare disease programs. Dr. Kravis receives no personal funds. Dr. Denny Porter through NICHD/NCATS has a cooperative research agreement with MNK. Dr. Porter personal financial disclosure

These organizations helped fund the cost of the PFDD meeting:

  • Mallinckrodt Pharmaceuticals
  • Orphazyme
  • StridBio
  • Together Strong Foundation

Disclosures: No company received and benefits from supporting the PFDD meeting and were not involved in the coordination of the meeting

Published: September, 2019.

Revisions and modifications: This document has not been revised and/or modified in any way after the version date listed above and on the cover page.

The submitters have all necessary permissions to submit this external resource and linking from the FDA website will not violate the proprietary rights of others.

Point of contact: Sean Kassen, skassen@nd.edu