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Scientific Advisory Board

As a national membership organization governed by parents and professionals, NNPDF fosters awareness, education, advocacy and research, supporting families and patients affected by Niemann-Pick disease. NNPDF’s unique position in the community is to remain unbiased in its education, advocacy and research efforts so that it serves the varying perspectives and needs of families and patients. Because of this mandate, NNPDF plays a critical role during the engagement of industry in clinical trials and throughout the federal regulatory process. Industry and the FDA depend upon national advocacy organizations that are governed to serve a wide range of patient and family needs. NNPDF is also the go to organization to augment scientific and clinical research, and to encourage professionals into the field. In order to properly support NNPDF’s mission in these areas, the Board has determined that the Scientific Advisory Board will  focus of advising NNPDF leadership with the research, clinical and regulatory leadership necessary to fulfill its role in the community.

The areas of responsibility for the SAB include providing expert advice and recommendations on:

  1. FDA regulatory issues
  2. Clinical issues
  3. Industry requests
  4. Annual conference topics and speakers related to research, clinical and regulatory
  5. Scientific and clinical materials that are utilized for public information for education
  6. NNPDF Board for future grant making fellowship program to promote clinical and research professionals to enter the field

Experts:  Research, neurology, clinical and regulatory in NPC, ASMD, related Lysosomal disorders
NNPDF Members:  Chair, Research/Clinical Board Member, Executive Director (non-voting)

Thank you to all of the members of our SAB who so generously offer their time and expertise to the NNPDF on a volunteer basis, without remuneration.


Lieberman 600
Andrew Lieberman, MD, PhD

NNPDF Scientific Advisory Board Chair
Professor, Department of Pathology
University of Michigan Medical School

Chrystelle Bougault 2021
Chrystelle Bougault

NNPDF Research/Clinical Board Member
Project Manager
UC San Diego Altman Clinical and Translational Research Institute

Hastings Caroline
Caroline Hastings, MD

Pediatric Hematologist-Oncologist
UCSF Benioff Children’s Hospital Oakland

Hopkin Justin
Justin Hopkin, MD

NNPDF Board Chair

Marc Patterson, MD

Professor of Neurology, Pediatrics & Medical Genetics
Chair, Division of Child and Adolescent Neurology
Mayo Clinic

Porter Denny
Forbes “Denny” Porter, MD, PhD

Senior Investigator & Clinical Director
National Institutes of Health

Schuchman Ed
Edward Schuchman, PhD

Genetic Disease Foundation – Francis Crick Professor of Genetics & Genomic Sciences
Vice Chair for Research, Department of Genetics & Genomic Sciences
Director, International Center for Types A & B Niemann-Pick Disease
Icahn School of Medicine at Mount Sinai

Sturley Steve
Stephen Sturley, PhD

Department of Biology
Barnard College, Columbia University

Wasserstein Melissa
Melissa Wasserstein, MD

Professor, Department of Pediatrics
Professor, Department of Genetics
Chief, Division of Pediatric Genetic Medicine, Department of Pediatrics
Montefiore Medical Center

Joslyn Crowe
Joslyn Crowe, MSW

NNPDF Executive Director (non-voting)

Supporting one another. Supporting our community.