Primary Clinicians / Researchers

Geneviève Bernard

Geneviève Bernard

Research Institute of the
McGill University Health Centre

Associate Professor, Departments of Neurology and Neurosurgery, Pediatrics and Human Genetics (associate member), McGill University;  Pediatric Neurologist, Department of Specialized Medicine, Division of Medical Genetics, McGill University Health Center;  Scientist, Child Health and Human Development, RI MUHC.

Adeline Vanderver

Adeline Vanderver

Children’s Hospital of Philadelphia

Program Director of the Leukodystrophy Center of Excellence at Children’s Hospital of Philadelphia (CHOP); Chair in Neurologic Disorders and Translational Neurotherapeutics at CHOP; founding member of the Global Leukodystrophy Initiative; Pediatric Neurologist and Geneticist.

vanderverlab.research.chop.edu

Nicole Wolf

Nicole Wolf

Amsterdam UMC

Child neurologist and clinical scientist at Amsterdam Leukodystrophy Center, working in the hypomyelinating leukodystrophy field since 2000. PI on hypomyelination and metachromatic leukodystrophy. Member of several editorial and scientific advisory boards.

4H Leukodystrophy Collaborative Network (#4HLCN)

Bernard Brais

Bernard Brais

Research Institute of the
McGill University Health Centre

Neurogeneticist of rare neurological
diseases.

Guangping Gaonderver

Guangping Gaonderver

UMass Medical School

Co-Director, Li Weibo Institute for Rare Diseases Research, Director, Horae Gene Therapy Center and Viral Vector Core, Professor of Microbiology and Physiological Systems, University of Massachusetts Medical School.

Benoit Coulombe

Benoit Coulombe

Institut de Recherches
Cliniques de Montréal

Director of the Department of Translational Proteomics at the Institut de Recherches Cliniques de Montréal and Professor at the Department of Biochemistry and Molecular Medicine, Université de Montréal.

Steve Goldman

Steve Goldman

University of Rochester Medical Center

URMC Distinguished Professor of Neuroscience and Neurology, and co-Director of Rochester’s Center for Translational Neuromedicine; Professor of Neuroscience at the University of Copenhagen in Denmark, co-director of the Center for Translational Neuromedicine, Professor of Neurology at Copenhagen University Hospital.

Joana Osorio

Joana Osorio

Center for Translational Neuroscience, University of Rochester Medical Center/USA and University of Copenhagen/Denmark

Child Neurologist (trained at the University of Pittsburgh Medical Center), 8-years experience in Cell Biology with focus in Cell and Gene Therapy in Steve Goldman’s lab, University of Rochester Medical Center and University of Copenhagen (PhD candidate).

Sébastien Fribourg

Sébastien Fribourg

INSERM U1212 – CNRS 5320

Structural biologist, with a research focus on RNA Polymerase III transcription initiation, pre-mRNA 3’-end processing and eukaryotic ribosome biogenesis.

Raphael Schiffman

Raphael Schiffman

Kimberly H. Courtwright and Joseph W. Summers Institute of Metabolic Disease

Medical Director of The Institute of Metabolic Disease at Baylor Research Institute, Dallas, Texas.

Paul Tesar

Paul Tesar

Case Western Reserve University
School of Medicine

Professor, with a research focus on stem cell pluripotency and differentiation, developmental neurobiology and genetics, regulation of glia, and therapeutic discovery.

Scientific Advisory Board

Walter Kowtoniuk

Walter Kowtoniuk

Third Rock Ventures

Father of two (and soon to be three), husband, and venture partner at Third Rock Ventures.

Florian Eichler

Florian Eichler

Massachusetts General Hospital
Harvard Medical School

Associate Professor of Neurology,
Director of the Center of Rare Neurological Diseases, Co-Founder of SwanBio Therapeutics.

Tom Barnes

Tom Barnes

Advisor

Advisor to the Yaya Foundation;
CEO Orna Therapeutics, Inc.