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NIH, Children's National Medical Center Team up on Rare Children's Disease Studies

NEW YORK (GenomeWeb News) – The National Institutes of Health and the NIH Clinical Center will partner with Children's National Medical Center to study and develop treatments for rare disorders, including projects that use genomics-based research approaches, NIH said on Tuesday.

Under the new Translational Research in Pediatrics Program, the collaborators will share resources and expertise, with Washington-based Children's National providing access to its staff as research partners for NIH investigators and access to its Clinical Research Center for outpatient visits, while NIH is providing scientists and research infrastructure.

The NIH Clinical Center sees more rare disease patients than any other center in the country, historically caring for those over two years old or weighing more than 20 pounds, NIH said.

The partners hope that some of their efforts will capitalize on new discoveries about the genetic causes of rare diseases and lead to new treatments that can be used earlier in the course of disease. The program is being piloted by the National Human Genome Research Institute.

The first of these collaborations is a study of young patients with methylmalonic academias, which affects between one in 50,000 and 80,000 babies born in the US. Charles Venditti, an investigator in NHGRI's Genetics and Molecular Biology Branch, and Kimberly Chapman, a principal investigator at the Children's Research Institute at Children's National, will lead the research.

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