Raredr

About

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 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 MedicineDivision of Endocrinology,
Metabolism, and Diabetes
University of Illinois at Chicago

Pat Furlong  
Founder and CEO of Parents Project MD

William Gannon Jr, MD 
Cheif Scientific Officer & Medical Director,
Capital City Technical Cunsulting

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

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 Allbama 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

Kari Luther Rosbeck
President & Chief Executive Officer, Tuberous Sclerosis Alliance

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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