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Academic Institutions

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  • Akron Children’s
  • Besta Institute
  • Binghamton University
  • Boston University
  • Brigham and Womens Hospital
  • Brighton and Sussex Medical School
  • Broad Institute
  • Case Western Reserve University
  • Charité University of Medicine, Berlin
  • Children’s Hospital of Philadelphia
  • Children’s National Health System
  • Children’s National Heart Institute
  • Cincinnati Children’s Hospital Medical Center
  • City University, London
  • Cleveland Clinic
  • Colorado State University
  • Columbia University
  • Diderot University Paris
  • Duke University
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  • Emory University 
  • Erciyes University
  • Federal University of Rio Grande do Sul
  • Gazi University
  • George Washington University
  • Hadassah Medical Center
  • Harvard University
  • Heidelberg University Hospital
  • Helmholtz Centre Munich
  • Houston Methodist Hospital
  • Hôpitaux Universitaires de Genève
  • Imperial College London
  • Indiana University School of Medicine
  • Johns Hopkins University
  • Karolinska University
  • Keio University
  • King’s College, London
  • Kyorin University

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  • Leiden University Medical Center
  • Lund University
  • Massachusetts General Hospital
  • Mayo Clinic
  • McGill University
  • Medical University of Innsbruck
  • Monash University
  • Mt Sinai
  • Nagoya University Hospital
  • Newcastle University
  • New York University
  • North Shore University Health System
  • Northwestern University
  • OULU Finland
  • Paris Brain Institute
  • Paris Descartes University-Necker Hospital
  • Queen Mary University of London
  • Radboud University
  • Showa University
  • Sorbonne University
  • Southern Illinois University
  • St. Marianna University
  • Stanford University
  • State Univ of New York – Buffalo
  • Stony Brook Medicine
  • Thomas Jefferson University
  • Tufts University
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  • UBonn Venusberg Campus

  • UMass Memorial

  • UNC Institute for Drug Safety Sciences

  • Univ College London Institute of Neurology

  • Univeristy of California San Francisco

  • University of Kansas
  • University College Cork, Ireland
  • University College London Hospital

  • University Hospital of Brussels

  • University Hospital of Liege

  • University of Alabama – Birmingham

  • University of Arizona

  • University of Arkansas

  • University of Birmingham, England

  • University of Bonn

  • University of Bristol

  • University of British Columbia

  • University of Calgary

  • University of California, Davis

  • University of California – Irvine

  • University of California – San Diego

  • University of California-San Francisco

  • University of Cambridge

  • University of Capetown

  • University of Chicago

  • University of Cologne

  • University of Colorado Denver

  • University of Florida

  • University of Genoa

  • University of Glasgow

  • University of Gothenburg

  • University of Hasslet

  • University of Helsinki

  • University of Illinois

  • University of Iowa

  • University of Leicester, UK

  • University of Leuven

  • University of Liverpool

  • University of Louisville

  • University of Lübeck

  • University of Manitoba

  • University of Maryland

  • University of Melbourne

  • University of Miami

  • University of Michigan

  • University of Minnesota

  • University of Missouri Kansas City 

  • University of Montreal

  • University of Munich

  • University of North Carolina

  • University of Oxford

  • University of Pennsylvania

  • University of Pittsburgh

  • University of Plymouth

  • University of Rochester Medical Center

  • University of Sheffield

  • University of Siena, Italy

  • University of Southern California

  • University of St. Andrews

  • University of Tampere

  • University of Texas

  • University of Toronto

  • University of Turku

  • University of Tübingen

  • University of Ulm

  • University of Utah

  • University of Virginia

  • University of Washington

  • University of Wurzburg

  • University of Zurich

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  • Vanderbilt University Medical Center

  • Virginia Commonwealth University

  • Washington University

  • Washington University

  • Western Washington University

  • Yale University

Pharmaceutical Companies

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  • Abbott Molecular

  • AbbVie

  • Acceleron

  • AceLink Therapeutics Inc

  • Actelion Pharmaceuticals Ltd

  • Aeglea BioTherapeutics, Inc.

  • Agios Pharmaceuticals Inc.

  • AKCEA Therapeutics

  • Alere

  • Allergan

  • Alnylam

  • Amgen, Inc

  • Annexon

  • Apellis Pharmaceuticals, Inc.

  • Applied Therapeutics

  • argenx

  • Astellas Pharma US, Inc.

  • AstraZeneca Pharmaceuticals LP

  • Audentes Therapeutics

  • Avidity Biosciences

  • AVROBIO

  • Baxter Healthcare Corporation

  • Bayer Pharma AG

  • Bayer US LLC

  • Becton Dickinson Diagnostic Systems

  • Biogen

  • Biohaven Pharmaceuticals, Inc.

  • BioMarin

  • BioMereiux

  • Biophytis

  • bluebird bio, Inc.

  • Boehringer Ingelheim Pharmaceuticals, Inc

  • Bridgebio

  • Bristol-Myers Squibb Company

  • Cabaletta Bio, Inc.

  • Calico Life Sciences

  • CareDx

  • Catabasis Pharmaceuticals

  • Celgene

  • Cepheid

  • Cerevel Therapeutics

  • Chiesi

  • CorrectorBio

  • Crown Bioscience, Inc

  • CSL Behring

  • CTI BioPharma

  • Cyclerion

  • Cydan Corporation

  • Daiichi Sankyo

  • Denali Therapeutics Inc.

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  • Edgewise Therapeutics
  • Eisai, Inc

  • Eli Lilly & Company

  • EMD Serono Research and Development Institute, Inc.

  • Entrada Therapeutics

  • Epirium Bio

  • EscapeBIo

  • Fulcrum Therapeutics

  • Galapagos NV

  • GE Healthcare

  • Genentech, Inc

  • Gilead Sciences

  • GlaxoSmithKline Corporation

  • Global BloodTherapeutics

  • Handl Therapeutics

  • Hansa Medical

  • Harmony Biosciences, LLC

  • Healx

  • Hoffman-La Roche, Inc

  • Horizon Pharma, Inc.

  • IMARA Therapeutics

  • Imbria Pharmaceuticals

  • Immucor, Inc.

  • imvaria

  • Infant Bacterial Therapeutics

  • Innate Immunotherapeutics

  • Inoviv

  • Integral Medicines

  • Ionis Pharmaceuticals, Inc.

  • Ironwood Pharmaceuticals, Inc
  • IXICO
  • Janssen Pharmaceutical Companies of Johnson & Johnson

  • Jazz Pharmaceuticals, Inc.

  • Johnson & Johnson Pharmaceutical Services

  • Kadmon Corporation

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  • LoQus23

  • Lundbeck

  • Lysogene

  • Magnus Therapeutics

  • Mallinckrodt Pharmaceuticals

  • MeiraGTx

  • Merck Sharp & Dohme Corporation

  • Metabolic Solutions Development

  • Millenium: The Takeda Oncology Company

  • MindMed, Inc.

  • Mironid

  • Mitsubishi Tanabe Pharma Corporation

  • Momenta Pharmaceuticals

  • Natera

  • Navitorpharma

  • Nephrogen

  • Nestlé Health Science

  • Neubase Therapeutics

  • NeuExcell Therapeutics

  • Neurametrix

  • Neurocrine Biosciences, Inc.

  • Neuroma

  • Novartis Pharmaceuticals Corporation

  • Novo Nordisk

  • NS Pharma

  • One Lambda, Inc.

  • OrbiMed

  • Otsuka

  • Ovid Therapeutics, Inc.

  • Oxford Brain Diagnostics

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  • PellaPharm, Inc.

  • Perspectum

  • Pfizer, Inc

  • PharmaStat

  • Prilenia

  • ProventionBio, Inc

  • PTC Therapeutics

  • Qiagen

  • Quantigen

  • Reata

  • Regeneron

  • REGENXBIO

  • Regulus Therapeutics

  • Relay Therapeutics

  • Roche

  • Sage

  • Sangamo Therapeutics

  • Sanofi-Aventis US, Inc

  • Santhera Pharmaceuticals, Inc.

  • Sarepta Therapeutics, Inc.

  • Sequella, Inc.

  • Servier Pharmaceuticals

  • Shire Pharmaceuticals, Inc

  • Sparrow Pharmaceuticals

  • SRI International

  • Summit (Oxford) Limited

  • Sunovion Pharmaceuticals, Inc.

  • Takeda Pharmaceuticals North America, Inc

  • Talaris Therapeutics

  • TEVA Pharmaceutical Industries, Ltd.

  • Thermofisher

  • Transplant Genomics

  • Triplet Tx

  • Trove Therapeutics

  • UCB Pharma

  • Ultragenyx Pharmaceutical

  • uniQure

  • Vaccinex

  • Veloxis

  • Vertex Pharmaceutical

  • VICO Therapeutics

  • Voyager Therapeutics

  • vTv Therapeutics

  • Wave Life Science

  • XORTX

Patient Groups

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    • Alzheimer’s Association

    • Alzheimer’s Drug Discovery Foundation

    • Alzheimer’s Research UK

    • Astarte Ventures

    • Ataxia Charlevoix-Saguenay Foundation

    • Ataxia UK

    • BLISS

    • CHDI Foundation

    • Congenital Muscle Disease International Registry

    • Cure CMD

    • CureDuchenne

    • Cure Parkinson’s Trust

    • Davis Phinney Foundation

    • Desmoid Tumor Research Foundation (DTRF)

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    • Epilepsy Foundation

    • European Huntington’s Disease Network

    • European Huntington Association

    • Families Blossoming

    • Foundation for Angelman Syndrome Therapeutics Australia

    • Foundation for Prader-Willi Research

    • Friedreich’s Ataxia Research Alliance

    • HNRNPH2-Related Disorders

    • Huntington’s Disease Society of America

    • Huntington’s Disease Youth Organization

    • Huntington Society of Canada

    • Huntington Study Group

    • International Niemann-Pick Disease Registry

    • International Parkinson and Movement Disorders Society
    • International Pemphigus and Perphigoid Foundation (IPPF)
    • Italian MS Society

    • JDRF

    • KHI Patient Family Partnership Council

    • March of Dimes

    • Michael J Fox Foundation

    • MitoAction

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    • National Ataxia Foundation

    • National MS Socieity of UK

    • National Multiple Sclerosis Society

    • National Multiple Sclerosis Society of Canada

    • National PKU Alliance

    • NEC Society

    • NICU Parent Network

    • Once Upon a Preemie

    • Parent Project Muscular Dystrophy

    • Parkinson’s Canada

    • Parkinson’s Disease Foundation

    • Parkinson’s UK

    • PKD Charity

    • PKD Foundation

    • PMD Alliance

    • Preemie World

    • Speaking for Moms and Babies, Inc.

    • Sturge-Weber Foundation

    • TSC Alliance

    • United Mitochondrial Disease Foundation (UMDF)

    • USAgainstAlzheimer’s

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