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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.



Andrew Lieberman, MD, PhD, Chair
Gerald D. Abrams Collegiate Professor of Pathology
Director of Neuropathology
University of Michigan Medical School

Andrew Lieberman, MD, PhD Web page

Conflict of Interest 2016 – A. Lieberman

Peter G Pentchev Ph D

Peter Pentchev, PhD
Emeritus Scientist
West Lafayette, IN

Dr. Pentchev Honored
at Family Conference

Jean Vance

Jean Vance, PhD
Professor, Division of Endocrinology
University of Alberta

Jean E. Vance, PhD Web page

Conflict of Interest 2016 – J. Vance


Daniel S. Ory, MD
Professor of Medicine
Cell Biology and Physiology
Washington University School of Medicine

Daniel S. Ory, MD Web page

Conflict of Interest 2016 – D. Ory


Fran Platt, PhD
Professor, Biochemistry and Pharmacology
University of Oxford

Fran Platt, PhD Web page

Conflict of Interest 2016 – F. Platt

Marie Vanier

Marie Vanier, MD, PhD
Research Director, INSERM
(French National Inst. of Health & Medical Research)

INSERM Web site

Conflict of Interest 2016 – M. Vanier


Marc C. Patterson, MD, 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, MD Web page

Conflict of Interest 2016 – M. Patterson

Forbes “Denny” Porter, MD, PhD
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, DVM, PhD
Professor, Department of Neuroscience
Albert Einstein College of Medicine

Steven U. Walkley,DVM, PhD Web page

Conflict of Interest 2016 – S. Walkley


Laura Liscum, PhD
Professor of Physiology
Tufts University School of Medicine

Laura Liscum, PhD Web page

Conflict of Interest 2016 – L. Liscum

Photo of Edward H. Schuchman

Edward H. 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

Edward H. Schuchman, PhD Web page

Conflict of Interest 2016 – E. Schuchman

Andrea Ballabio

Andrea Ballabio, MD
Scientific Director
TIGEM, Telethon Institute of Genetics and Medicine

Andrea Ballabio, MD Web page

Stephen L. Sturley, Ph.D.

Stephen Sturley, PhD
Associate Professor of Clinical Nutrition
Institute of Human Nutrition
Columbia University

Stephen Sturley, PhD Web page

Conflict of Interest 2016 – S. Sturley

Heiko Runz

Heiko Runz, MD
Specialist in Medical Genetics
University of Heidelberg, Germany

Heiko Runz, MD Web page

Conflict of Interest 2016 – H. Runz


Jean Gruenberg, PhD
Professor of Biochemistry
University of Geneva – Sciences II

Jean Gruenberg, PhD Web page


Lola Ledesma, PhD
Centro Biologia Molecular Severo
Ochoa, Madrid, Spain

Lola Ledesma, PhD Web page

Conflict of Interest 2016 – L. Ledesma

Vite Photo

Charles H. Vite, DVM, PhD
Associate Professor, Neurology School of Veterinary Medicine, University of Pennsylvania

Charles H. Vite, DVM, PhD Web page

Conflict of Interest 2016 – C. Vite

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

Dr. Robert J. Desnick

Robert J. Desnick, MD, PhD

Dean for Genetic and Genomic Medicine
Professor and Chair Emeritus
Mount Sinai School of Medicine

Robert J. Desnick, MD, PhD Web page

Yiannis Ioannou, Ph.D.

Yiannis A. Ioannou, PhD
Associate Professor, Genetics & Genomic Science
Mount Sinai School of Medicine

Yiannis A. Ioannou, PhD Web page

02/02/2017 jjb