What's New
JULY 9, 2012
PRESS RELEASE ~ Barth Syndrome Foundation selected for Global Rare Disease Registry
FOR IMMEDIATE RELEASE:
Barth Syndrome Foundation selected for Global Rare Disease Registry – July 9, 2012
Barth Syndrome Foundation (BSF) has been selected as one of thirty-four rare disease organizations to participate in a two-year pilot program of the National Institutes of Health called the Global Rare Disease Registry and Data Repository (GRDR). As a pilot participant, BSF will work in partnership with leaders in rare disease research at the Office of Rare Diseases Research, National Center for Advancing Translational Sciences, National Institutes of Health, PatientCrossroads, Children Hospital of Philadelphia and WebMD. The GRDR program will collect de-identified patient health information from participating registries established by the individual rare disease organizations in order to allow analyses of data across many rare diseases as well as to facilitate clinical trials and other studies.
“We are delighted to take part in the pilot program, as research that we have funded for over a decade has made it clear that Barth syndrome, as a multi-system disorder, shares aspects of many other diseases. We want to be part of research across diseases and broader clinical trials that might ultimately help affected individuals,” said BSF Chair, Steve McCurdy.
BSF believes that a collaborative approach is the best way to serve families and, ultimately, to find a specific treatment. Since 2002 BSF has funded 63 peer-reviewed research grants totaling over $2.3 million, hosts a biennial scientific, medical and family conference, maintains a genetic mutation database for the causative gene of Barth syndrome (tafazzin). We currently have affiliates in Canada, France, the United Kingdom and South Africa.
BSF (www.barthsyndrome.org) is dedicated to supporting those with Barth syndrome, their medical professionals, and the scientists who work to understand the disorder. “The Barth community has benefitted from the research that has emerged in the last decade, and we expect the GRDR effort to attract even more efforts to test scientific or clinical theories. We will never give up on our mission and want to help in any way we can,” Mr. McCurdy declared.
Information regarding the Global Rare Disease Registry and Data Repository pilot program is available at http://grdr.info.
Contact:
Lindsay Groff, MBA, Executive Director
Barth Syndrome Foundation
lindsay.groff@barthsyndrome.org
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