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Scientific Advisory Board

Scientific Advisory Board (SAB)

The Scientific Advisory Board (SAB) gives guidance to the NNPDF’s Board of Directors in determining policies and funding decisions related to research. The commitment and guidance of the SAB has been critical to our success thus far, and we greatly appreciate all their hard work on behalf of families affected by NPD.

SAB members evaluate grant and fellowship funding applications to determine where research funds may best be applied as we look to accelerate the pace toward effective treatments and a cure for all types of Niemann-Pick Disease.

Members of the SAB also attend the annual NNPDF Family Support and Medical Conference to give presentations and update families on the latest research developments. They also give generously of their time to talk with families and answer questions families may have relating to clinical and research matters.

Specialties of SAB members are wide-ranging and include pediatric neurology, genetics, physiology, human nutrition, metabolism and cholesterol trafficking, molecular and cell biology, biochemistry, etc. (To learn more about the work of individual SAB members, visit the links below the photos.)

We are indebted 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.

Thank you, SAB!


FRONT ROW: Marie Vanier, Jean Vance, Laura Liscum, Lola (Delores) Ledesma
MIDDLE ROW: Forbes “Denny” Porter, Andrew Lieberman, Daniel Ory, Edward Schuchman
BACK ROW: Steven Walkley, Charles Vite, Stephen Sturley, Heiko Runz

NNPDF Scientific Advisory Board


The members of the NNPDF Scientific Advisory Board have asked us to forward to the NNPDF Membership their policy governing transparency and conflicts of interest. This policy was unanimously approved by the entire SAB, an international group of 17 researchers and clinician-scientists. Click below for the Conflict of Interest Policy adopted by the Scientific Advisory Board as of August 2016.

SAB Conflict of Interest Policy

NNPDF Scientific Advisory Board


SAB Meeting Minutes | August 11, 2016


Andrew Lieberman, M.D., Ph.D., Chair
Gerald D. Abrams Collegiate Professor of Pathology
Director of Neuropathology
University of Michigan Medical School
Andrew Lieberman, M.D., Ph. D. Web page

Conflict of Interest 2016 – A. Lieberman

Peter G Pentchev Ph D

Peter Pentchev, Ph.D.
Emeritus Scientist
West Lafayette, IN
Dr. Pentchev Honored
at Family Conference

Jean Vance

Jean Vance, Ph.D
Professor, Division of Endocrinology
University of Alberta
Jean E. Vance, Ph.D. Web page

Conflict of Interest 2016 – J. Vance


Daniel S. Ory, M.D.
Professor of Medicine
Cell Biology and Physiology
Washington University School of Medicine
Daniel S. Ory, M.D. Web page

Conflict of Interest 2016 – D. Ory


Fran Platt, Ph.D.
Professor, Biochemistry and Pharmacology
University of Oxford
Fran Platt, Ph.D. Web page

Conflict of Interest 2016 – F. Platt

Marie Vanier

Marie Vanier, M.D., Ph.D.
Research Director, INSERM
(French National Inst. of Health & Medical Research)
INSERM Web site

Conflict of Interest 2016 – M. Vanier


Marc C. Patterson, M.D., FRACP Chair, Division of Child and Adolescent Neurology; Professor of Neurology, Pediatrics & Medical Genetics; Director, Child Neurology Training Program; Mayo Clinic Children’s Center; Mayo Clinic, Minnesota
Marc Patterson, M.D. Web page

Conflict of Interest 2016 – M. Patterson

Forbes “Denny” Porter, M.D., Ph.D.
Senior Investigator,
Section on Molecular Dysmorphology, Program in Developmental Endocrinology and Genetics
Natl. Institute of Child Health & Human Development
Natl. Institutes of Health
NICHHD Intramural Research Web page

Conflict of Interest 2016 – F. Porter

Dr. Steven U. Walkley

Steven U. Walkley, D.V.M., Ph.D.
Professor, Department of Neuroscience
Albert Einstein College of Medicine

Steven U. Walkley,DVM, Ph.D. Web page

Conflict of Interest 2016 – S. Walkley


Laura Liscum, Ph.D.
Professor of Physiology
Tufts University School of Medicine
Laura Liscum, Ph.D. Web page

Conflict of Interest 2016 – L. Liscum

Photo of Edward H. Schuchman

Edward H. Schuchman, Ph.D.
Genetic Disease Foundation-Francis Crick Professor
Vice Chairman for Research
Professor of Gene and Cell Medicine
Mount Sinai School of Medicine
Edward H. Schuchman, Ph.D. Web page

Conflict of Interest 2016 – E. Schuchman

Andrea Ballabio

Andrea Ballabio, M.D.
Scientific Director
TIGEM, Telethon Institute of Genetics and Medicine
Andrea Ballabio, M.D. Web page

Stephen L. Sturley, Ph.D.

Stephen Sturley, Ph.D.
Associate Professor of Clinical Nutrition
Institute of Human Nutrition
Columbia University
Stephen Sturley, Ph.D. Web page

Conflict of Interest 2016 – S. Sturley

Heiko Runz

Heiko Runz, M.D.
Specialist in Medical Genetics
University of Heidelberg, Germany
Heiko Runz, M.D. Web page

Conflict of Interest 2016 – H. Runz


Jean Gruenberg, Ph.D
Professor of Biochemistry
University of Geneva – Sciences II
Jean Gruenberg, Ph.D Web page


Lola Ledesma, Ph.D
Centro Biologia Molecular Severo
Ochoa, Madrid, Spain
Lola Ledesma, Ph.D Web page

Conflict of Interest 2016 – L. Ledesma

Vite Photo

Charles H. Vite, DVM, Ph.D.
Associate Professor, Neurology School of Veterinary Medicine, University of Pennsylvania
Charles H. Vite, DVM, Ph.D. Web page

Conflict of Interst 2016 – C. Vite

Esteemed Former SAB Members — Thank you for your dedicated service!

Dr. Robert J. Desnick

Robert J. Desnick, M.D., Ph.D.
Professor, Department of Human Genetics
Mount Sinai School of Medicine
Robert J. Desnick, M.D., Ph. D. Web page

Yiannis Ioannou, Ph.D.

Yiannis A. Ioannou, Ph.D.
Associate Professor, Genetics & Genomic Science
Mount Sinai School of Medicine
Yiannis A. Ioannou, Ph. D. Web page

09/08/2016 jjb