Primary Clinicians / Research Partners

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

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.

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

Research Institute of the McGill University Health Centre

Neurogeneticist of rare neurological
diseases; Co-director of the Rare Neurological Diseases group of the Montreal Neurological Institute and Hospital; Co-director of McGill’s Neuromuscular and Neurogenetics Fellowships;
Full Professor of Neurology and Human Genetics at McGill University in Montreal.

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.

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.

Guangping Gao

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.

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

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

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).

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

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.

Jun Xie

Vector Core

Director, Vector Development of Vector Core; Associate Professor; Horae Gene Therapy Center; Department of Microbiology & Physiological Systems; University of Massachusetts Chan Medical School

Scientific Advisory Board

Tom Barnes


CEO Orna Therapeutics, Inc., and Executive Partner at MPM Capital

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.

Walter Kowtoniuk

Third Rock Ventures

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

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