Rare Disease Forum at Whitehead Institute

Thursday, February 27 2014
5:30 pm – 6:30 pm

Location: Whitehead Institute, 9 Cambridge Center, Cambridge, MA 02142

2014 Rare Disease Forum: “Curing Rare Childhood Diseases: New Industry and Academic Models”
Dress: Casual
Contact: Lauren Gilbert at (617) 258-5489 or lgilbert@wi.mit.edu

In recognition of International Rare Disease Day, we are delighted to host the 3rd annual Rare Disease Forum at Whitehead Institute. Join us for this special event on Thursday, February 27th at 5:30 pm to hear from leaders in rare and orphan disease research in the Boston area.

This year’s forum will investigate “Curing Rare Childhood Diseases: New Industry and Academic Models” and will feature Whitehead Member Harvey Lodish, Jeffrey Leiden, President and CEO, Vertex Pharmaceuticals, Alan Beggs, Director of The Manton Center for Orphan Disease Research, and David Williams, Chief, Division of Hematology/Oncology and Director of Translational Research, Boston Children’s Hospital.

Following the discussion, Whitehead is hosting a networking session where attendees will have the opportunity to discuss the various challenges and opportunities faced in the advocacy and promotion of rare disease research. We are pleased to be partnering with several rare disease advocacy organizations, noted below, this year.

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ABOUT OUR SPEAKERS

Alan Beggs, PhD
Director, The Manton Center for Orphan Disease Research

Alan Beggs is director of The Manton Center for Orphan Disease Research at Children’s Hospital Boston and the Sir Edward and Lady Manton Professor of Pediatrics at Harvard Medical School. He received his AB in biology at Cornell University and his PhD in human genetics at Johns Hopkins University. He completed postdoctoral fellowships in medical genetics at Johns Hopkins University and in clinical molecular genetics at Harvard Medical School, and has directed an independent research laboratory in the Genetics Division at Boston Children’s Hospital since 1992.  Dr. Beggs has led studies to identify the genetic basis for a wide variety of rare conditions with a particular focus on skeletal muscle and its defects in congenital myopathies and related neuromuscular diseases.  Current research is focused on developing next generation sequencing tools for clinical practice and on utilizing genetic approaches in human patients and animal models to understand the pathophysiology and to develop targeted therapies to treat these devastating disorders of childhood.

Jeffrey Leiden, MD, PhD
Chair, President and CEO, Vertex Pharmaceuticals

Jeffrey Leiden joined Vertex’s executive team in December 2011, and has served as a member of Vertex’s board of directors since 2009. Dr. Leiden brings to Vertex more than 20 years of scientific, commercial and financial experience in the pharmaceutical and biotechnology industries and clinical experience in academia as a practicing cardiologist and molecular biologist. Dr. Leiden began his career in academia as a practicing cardiologist and molecular biologist. From 1987 to 2000, Dr. Leiden held several academic appointments, including roles as Chief of Cardiology at the University of Chicago and Professor of Medicine at Harvard Medical School and Brigham and Women’s Hospital. During his academic career, Dr. Leiden was also involved in starting several biotechnology companies including Vical and Cardiogene. Dr. Leiden is an elected member of both the American Academy of Arts and Sciences, and the Institute of Medicine of the National Academy of Sciences. Dr. Leiden received his M.D., Ph.D. and B.A. degrees from the University of Chicago.

Harvey Lodish, PhD
Whitehead Member

Whitehead Founding Member Harvey Lodish’s research areas include red blood cell development, hematopoietic stem cells, and the regulatory roles microRNAs play in the development and function of muscle cells, fat cells, and blood cells. Dr. Lodish’s latest work on microRNAs has shed new light on our understanding of fat cells, resulting in important implications for the treatment of obesity and diabetes.  Dr. Lodish joined the MIT faculty in 1968. He has been a professor of biology since 1976 and professor of bioengineering since 1999. He earned his PhD at Rockefeller University in 1966. He was elected a fellow of the American Association for the Advancement of Science in 1986, a member of the National Academy of Sciences in 1987, and a fellow of the American Academy of Arts and Sciences in 1999. He is a member of the Board of Trustees of Boston Children’s Hospital, and is Chair of the Scientific Advisory Board of the Massachusetts Life Sciences Center, charged with oversight of the state’s 10- year, $1 billion investment in the life sciences. He is also the lead author of the textbook Molecular Cell Biology.

David Williams, MD
Chief, Division of Hematology/Oncology and Director of Translational Research, Boston Children’s Hospital

Dr. Williams is the Chief of Hematology/Oncology and Director of Translational Research at Boston Children’s Hospital and Associate Chairman, Department of Pediatric Oncology at Dana-Farber Cancer Institute. He is Director of the Pediatric Hematology/Oncology Fellowship Training Program at BCH/DFCI. After completing his post-doctoral fellowship at MIT Cancer Center and the Whitehead Institute, he was a Howard Hughes Medical Institute Investigator for 16 years and his laboratory has been continuously NIH funded since 1986. His basic research has focused on hematopoietic stem cell biology, including genetic diseases of the blood and specifically molecular and biochemical analysis of the interaction between hematopoietic stem cells and the bone marrow supporting environment.

About Rare Disease Day

Rare Disease Day is an annual, awareness-raising event coordinated by EURORDIS at the international level and the National Alliances of Patient Organisations at the national level. The main objective of Rare Disease Day is to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients’ lives. February 2014 marks the sixth international Rare Disease Day.

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