Rare Disease Report


Rare Disease Report is a website and weekly e-newsletter that offers an independent voice for the Rare Disease Community. It strives to bring together medical, scientific, investment, regulatory, and advocate professionals interested in rare diseases and orphan drugs.

Our Team includes:
Krista Rossi
Assistant Editor

Our Advisory Board includes:

Peter C. Adamson, MD
Chair, Children's Oncology Group
Chief, Division of Clinical Pharmacology & Therapeutics
The Children's Hospital of Philadelphia
Christie Ballantyne, MD
Professor, Molecular and Human Genetics, Baylor College of Medicine
Nicole Boice
Founder & CEO, Global Genes
Kyle Brown
CEO and founder of PatientCrossroads
Lucie Bruijn, PhD
Science Director at Amyotrophic Lateral Sclerosis Association
Barbara K Burton, MD
Director, PKU Program; Professor of Pediatrics,
Northwestern University Feinberg School of Medicine
Mark Cannon, DDS
Professor, Clinical Otolarynology Dental Surgery
Shan Chandrakasan, MD
Cincinnati Children’s Medical Center
Joshua P. Cohen, PhD
Senior Research Fellow/ Research Assistant Professor.
Tufts Center for the Study of Drug Development
Timothy Craig, DO 
Allergy, Immunology and Respiratory Diseases, Penn State Hershey Medicine
Lawrence A. Frohman, MD
Professor Emeritus of Medicine, Division of Endocrinology,
Metabolism, and Diabetes, University of Illinois at Chicago
Pat Furlong 
Founder and CEO of Parents Project MD
William Gannon Jr, MD
Chief Scientific Officer & Medical Director,
Capital City Technical Consulting
Guillermo Garcia-Manero, MD
Professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center
Audrey Gordon
President, Progeria Research Foundation
Eric Hoffman, PhD
ReveraGen Biopharma; Vice President, AGADA Biosciences; Director, Center for Genetic Medicine Research, Children's National Medical Center; Professor and Chair, Department of Integrative Systems Biology, George Washington University
Melissa Hogan
Founder, Saving Case
Jeffrey Jones, MD
Assistant Professor of Internal Medicine, Ohio State University
Wahida Karmally, DrPH, RD, CDE, CLS, FNLA
Irving Institute for Clinical and Translational Research, Columbia University
Thomas Kirkegaard Jensen, PhD
Chief Scientific Officer, Orphazyme ApS
Abhishek Kumar, MD
Hematology Oncology, New York Medical College-St. Joseph’s Regional Medical Center
Michael Levy, MD, PhD 
Assistant Professor, Johns Hopkins University
Medical Director of General Neurology, Johns Hopkins Hospital
Phillip A Low, MD
Professor of Neurology, Mayo Clinic
Kari Luther Rosbeck
President & Chief Executive Officer, Tuberous Sclerosis Alliance

Pam Mace, RN
FMD Patient and Executive Director, Fibromuscular Dysplasia Society of America
Brian Malkin 
McGuireWoods LLP; Co-editor www.fdalife.com
Jay (John T.) Matthews, MBA, PhD
Associate Director
Global Clinical Trial Operations, The Americas | Merck Research Laboratories
John Mascarenhas, MD
Associate Professor, Medicine, Hematology and Medical Oncology, Mount Sinai Hospital
Nikhil Munshi, MD
Professor of Medicine, Harvard Medical School
Brian P. O'Sullivan, MD
Professor of Pediatrics;  Director, Cystic Fibrosis Center; Chair,
Institutional Review Board; Associate Vice-Provost for Research, UMass Medical School
Shin Oh, MD
Neurologist, University of Alabama Birmingham
Marlana Orloff, MD
Oncologist, Thomas Jefferson University
Amy Paller, MD
Chair of Dermatology, Professor of Pediatrics, Northwestern University Feinberg School of Medicine
Ethan Perstein, PhD
Founder and CEO, Perlstein Lab
Javier M Romero, MD
Director of Ultrasound, Associate Director of Neurovascular Laboratory, Massachusetts General Hospital
H Diana Rosas, MD
Associate Professor Neurology, Harvard Medical School; Neurologist, Massachusetts General Hospital;
Director, Center for Neuro-imaging of Aging and Neurodegenerative Disease.
Yaffa Rubinstein, PhD
Director of Patient Resources for Clinical and Translational Research,
Office of Rare Diseases Research (ORDR), National Center for Advancing Translational Sciences (NCATS). National Institutes of Health
Phillip W Ruff PhD, CChem, MRSC
Director, Global Market Access, Shire
Scott Shirley
Executive Director, Uplifting Athletes
Dean Suhr
President and Co-founder, MLD Foundation; Registry Manager, Genetic Alliance; rare disease advocate and consultant
Jitske Tiemensma
Assistant Professor, University of California, Merced
Shunji Tomatsu MD, PhD
Director, Skeletal Dysplasia Lab,  Departments of Biomedical Research and Orthopedics, 
ARB 331 Nemours/Alfred I. duPont Hospital for Children
Clara van Karnebeek MD, PhD, FCCMG
Assistant Professor Biochemical Genetics & Pediatrics, University of British Columbia, Vancouver Canada
Louise Vetter
CEO of Huntington’s Disease Society of America.
James Underberg, MD
Director, Bellevue Hospital Primary Care Lipid Management Clinic, NYC
Neal J Weinreb, MD, FACP
Voluntary Associate Professor of Medicine, Dr John A Macdonald Foundation Department of Human Genetics, University of Miami Miller School of Medicine.
Director, University Research Foundation for Lysosomal Storage Diseases Inc, Coral Springs and Boca Raton, Florida, USA
James Wheless, MD
Professor and Chief, Department of Pediatric Neurology, University of Tennessee Health Science Center
Don Wilson, MD
Physician of Pediatric Endocrinology, Cooks Children's Physician Network


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