Children's Hospital Boston Receives $25 Million to Study Rare Diseases in Children
Children's Hospital Boston has announced a $25 million grant from the New York City-based Manton Foundation to establish what is believed to be the world's first center for the study of rare, or "orphan," diseases.
The Manton Center for Orphan Disease Research will provide support to scientists from Children's Hospital and Harvard Medical School working to develop new diagnoses and treatments for rare diseases, including genetic syndromes, immune system problems, and metabolic and neuromuscular disorders. These illnesses affect mostly children, often under the age of two, and occur too infrequently — fewer than 200,000 cases in the United States — to attract the resources and research devoted to more common diseases. There are 6,000 known rare diseases, and half of them cause motor, sensory, or intellectual deficiencies, while another 20 percent cause chronic pain.
The gift also will establish a new chair at Harvard — the Sir Edwin and Lady Manton Professorship in Pediatrics in the field of genetics — to be held by Alan H. Beggs, who also will serve as director of the Manton Center. The facility will support the research of seven senior scientists, including Beggs. In addition, the gift will provide support for a gene discovery core in which patients' genetic information is collected from tissue samples; innovation awards for junior faculty; two or three research fellowships; and a visiting scientist program.
"For years, orphan disease research has been hindered by a lack of resources and attention," said Children's Hospital CEO Dr. James Mandell. "This new center will open a superhighway for discovery, which we believe will lead not only to insights into the biological underpinnings of various diseases but also to better diagnoses and new treatments for children and adults worldwide."
