Who We Are
Who We Are
PATIENTS
CAREGIVERS
RESEARCHERS
CLINICIANS
INDUSTRY
NONPROFITS
The N=1 Collaborative is a global community of researchers, clinicians, drug developers, patient advocates, and regulators working to advance the field of individualized medicines.
We are a global society working together to advance individualized medicines for ultra-rare diseases by developing an end-to-end pathway that is rigorous, reproducible, and openly available to everyone.
Board of Directors
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Julia Vitarello
Mila’s Mom
Mila’s Miracle Foundation
Winston Yan, M.D., Ph.D.
Founding President, N=1 Collaborative
Director, Center for Genetic Surgery (CGS), Broad Institute
Tim, Yu, M.D., Ph.D.
Boston Children’s Hospital
Harvard Medical School
Board of Directors
Click here to read full bios
Julia Vitarello
Mila’s Mom
Mila’s Miracle Foundation
Winston Yan, M.D., Ph.D.
Founding President, N=1 Collaborative
Director, Center for Genetic Surgery (CGS), Broad Institute
Tim, Yu, M.D., Ph.D.
Boston Children’s Hospital
Harvard Medical School
Nicole Boice
Co-Founder, SEE RARE™
Founder, Global Genes & RARE-X
Holm Graessner
Managing Director, Centre for Rare Diseases, University Hospital Tübingen, Germany
Brian Tseng
The POLG Foundation
Nicole Boice
Co-Founder, SEE RARE™
Founder, Global Genes & RARE-X
Holm Graessner
Managing Director, Centre for Rare Diseases, University Hospital Tübingen, Germany
Brian Tseng
The POLG Foundation
Scientific Advisory Board
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Annemieke Aartsma-Rus, Ph.D.
Professor of Translational Genetics
Dutch Center for RNA Therapeutics (DCRT)
Leiden University Medical Center, Department of Human Genetics
Leiden, The Netherlands
Scott Demarest, MD MSCS
Chief Precision Medicine Officer, Precision Medicine Institute, Children’s Hospital Colorado
Associate Professor, Departments of Pediatrics and Neurology, University of Colorado School of Medicine
Richard Finkel, M.D.
Director of Center of Experimental Neurotherapeutics
St. Jude Children’s Research Hospital
Ashley Kuniholm
Regulatory Affairs Manager
Boston Children’s Hospital
Harvard University
Marlen Lauffer, M.D.
Senior Researcher
Dutch Center for RNA Therapeutics (DCRT), Leiden University Medical Center, The Netherlands
Erin McConnell, Ph.D.
Research Scientist
Carleton University
Matthis Synofzik, MD
Professor of Neurology, Head of Division, Research Division of Translational Genomics of Neurodegenerative Diseases, Hertie-Institute for Clinical Brain Research & Center of Neurology, University of Tübingen, 1 Mutation 1 Medicine (1M1M)
Bryan J. Traynor, M.D., Ph.D.
Senior Investigator
National Institute of Aging
Olivia Kim-McManus M.D.
UCSD Associate Professor of Neurosciences, Rady Children’s Institute for Genomic Medicine
Jillian Belgrad
M.D./Ph.D Candidate, MS3, Khvorova Lab, RNA Therapeutics Institute, UMass Chan Medical School
Peter Oliver, PhD
Head of Research, Nucleic Acid Therapy Accelerator
Associate Professor of Neuroscience, University of Oxford
Roger J Paxton, PhD
Research Manager, Precision Medicine Institute, Children’s Hospital Colorado
Stephan Sanders
Professor of Paediatric Neurogenetics, Department of Paediatrics, University of Oxford
Willeke van Roon-Mom, PhD
Professor Translational Studies of Neurodegenerative Disorders, Department of Human Genetics, Leiden University Medical Center
Tobias Pöhlmann
CEO, XNApharma
Professor for Biomaterials, University of Applied Science in Zwickau, Germany
Margaret Meserve, M.S., CGC
Research Genetic Counselor, Boston Children’s Hospital
Winston Yan, M.D., Ph.D.
Founding President, N=1 Collaborative
Director, Center for Genetic Surgery (CGS), Broad Institute
Tim, Yu, M.D., Ph.D.
Boston Children’s Hospital
Harvard Medical School
Hélène Tran
Research Director, Neurology Therapeutic Area, Servier Research & Development Institute
Seppe Hermans
PhD Candidate, Technische Universität Ilmenau (TU Ilmenau / Technical University of Ilmenau), BianoGMP GmbH, Medicine Made to Measure
Tea Umek, PhD
Researcher, Karolinska University Hospital, Karolinska Institute
Margot A. Cousin, PhD
Assistant Professor of Medical Genetics
Director, N-of-1 Therapeutics Program, Mayo Clinic Center for Individualized Medicine, Department of Neurology, Department of Molecular Medicine, Mayo Clinic
Scientific Advisory Board
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Annemieke Aartsma-Rus, Ph.D.
Professor of Translational Genetics
Dutch Center for RNA Therapeutics (DCRT)
Leiden University Medical Center, Department of Human Genetics
Leiden, The Netherlands
Scott Demarest, M.D.
Assistant Professor of Pediatrics and Neurology
Precision Medicine Institute, Children’s Hospital Colorado
University of Colorado School of Medicine
Richard Finkel, M.D.
Director of Center of Experimental Neurotherapeutics
St. Jude Children’s Research Hospital
Ashley Kuniholm
Regulatory Affairs Manager
Boston Children’s Hospital
Harvard University
Marlen Lauffer, M.D
IDRM Transition Research Fellowship, Lab Head, Institute of Developmental and Regenerative Medicine, University of Oxford, UK
Erin McConnell, Ph.D.
Research Scientist
Carleton University
Matthis Synofzik, MD
Professor of Neurology, Head of Division, Research Division of Translational Genomics of Neurodegenerative Diseases, Hertie-Institute for Clinical Brain Research & Center of Neurology, University of Tübingen, 1 Mutation 1 Medicine (1M1M)
Bryan J. Traynor, M.D., Ph.D.
Senior Investigator
National Institute of Aging
Olivia Kim-McManus M.D.
UCSD Associate Professor of Neurosciences, Rady Children’s Institute for Genomic Medicine
Jillian Belgrad
M.D./Ph.D Candidate, MS3 Khvorova Lab, RNA Therapeutics Institute, UMass Chan Medical School
Peter Oliver, PhD
Head of Research, Nucleic Acid Therapy Accelerator
Associate Professor of Neuroscience, University of Oxford
Stephan Sanders
Professor of Paediatric Neurogenetics, Department of Paediatrics, University of Oxford
Roger J Paxton, PhD
Research Manager, Precision Medicine Institute, Children’s Hospital Colorado
Willeke van Roon-Mom, PhD
Professor Translational Studies of Neurodegenerative Disorders, Department of Human Genetics, Leiden University Medical Center
Tobias Pöhlmann
CEO, XNApharma
Professor for Biomaterials, University of Applied Science in Zwickau, Germany
Margaret Meserve, M.S., CGC
Research Genetic Counselor, Boston Children’s Hospital
Winston Yan, M.D., Ph.D.
Founding President, N=1 Collaborative
Director, Center for Genetic Surgery (CGS), Broad Institute
Tim, Yu, M.D., Ph.D.
Boston Children’s Hospital
Harvard Medical School
Operations Team
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Nicole Nolen
Executive Director
N=1 Collaborative
Nina Green
Senior Advisor to the Vice President, Technology Innovation and Development Office
Boston Children’s Hospital
Harvard University
Ashley Kuniholm
Regulatory Affairs Manager
Boston Children’s Hospital
Harvard University
Operations Team
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Nicole Nolen
Executive Director
N=1 Collaborative
Nina Green
Senior Advisor to the Vice President, Technology Innovation and Development Office
Boston Children’s Hospital
Harvard University
Ashley Kuniholm
Regulatory Affairs Manager
Boston Children’s Hospital
Harvard University
Erin McConnell, Ph.D.
Research Scientist
Carleton University
Margaret Meserve, M.S., CGC
Research Genetic Counselor, Boston Children’s Hospital
Melody Joy Paine
N1C Corporate Alliance Lead, N=1 Collaborative
Founder & Storyteller Rare Rebels™, Imperfect JOY®
Emily Sparago
Corporate Alliance Coordinator
N=1 Collaborative
Winston Yan, M.D., Ph.D.
Founding President, N=1 Collaborative
Director, Center for Genetic Surgery (CGS), Broad Institute
Tim, Yu, M.D., Ph.D.
Boston Children’s Hospital
Harvard Medical School
Erin McConnell, Ph.D.
Research Scientist
Carleton University
Margaret Meserve, M.S., CGC
Research Genetic Counselor, Boston Children’s Hospital
Melody Joy Paine
N1C Corporate Alliance Lead, N=1 Collaborative
Founder & Storyteller Rare Rebels™, Imperfect JOY®
Emily Sparago
Corporate Alliance Coordinator
N=1 Collaborative
Winston Yan, M.D., Ph.D.
Founding President, N=1 Collaborative
Director, Center for Genetic Surgery (CGS), Broad Institute
Tim, Yu, M.D., Ph.D.
Boston Children’s Hospital
Harvard Medical School
Workgroups
Our workgroups have rolling enrollment so we keep the list of members up-to-date in Connections, which you can access HERE.
Workgroups
Our workgroups have rolling enrollment so we keep the list of members up-to-date in Connections, which you can access HERE.