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Scientific and Medical Conference about Barth Syndrome

Award Number: R13HL124706
ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 06/25/2014
PERIOD OF PERFORMANCE END DATE: 05/31/2015

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Scientific and Medical Conference about Barth Syndrome - DESCRIPTION (provided by applicant): The specific aims of this international conference are to identify and suggest ways to overcome those clinical and scientific challenges that are hindering progress in finding effective clinical therapies specific for Barth syndrome (BTHS) individuals. This conference will address these challenges by: 1) bringing together the physicians, healthcare providers, and scientists working on the biochemical, genetic, and clinical questions about BTHS to discuss their work; 2) increasing the number of researchers and physicians involved with BTHS or BTHS individuals; 3) fostering interdisciplinary discussion, promoting working collaborations, and setting the agenda for advancing specific therapies for BTHS; 4) providing a forum to evaluate these specific therapies; and 5) promoting the communication and publication of BTHS related material. Previous conferences in this series have heralded significant scientific advances, but what remains elusive is the clinical translation of what we know about the dysfunction of the tafazzin gene (the gene linked with BTHS) into specific therapies. Though progress has been made since the last Conference, a more comprehensive understanding of how tafazzin dysfunction leads to the morbidity and mortality is still needed. Invigorated by these biennial exchanges of scientific/medical information, for 2014 the BTHS research community can now point to and discuss in more detail the following clinically-relevant accomplishments/studies: the orphan- drug designation of bezafibrate, the plans for clinical studies with bezafibrate and resveratrol, nutritional therapy, enzyme replacement therapy, cardiolipin replacement therapy, screening of pharmacological compounds in a high throughput format, and the completion of two exercise clinical studies. I believe that these biennial Conferences have accelerated the timeframe of clinically important advancements as well as scientific accomplishments by directly encouraging the researchers involved. These meetings bring together the key scientists and clinicians who can translate the accumulated research knowledge into potential treatments, and just as critically, who also can evaluate these treatments in the context of the life-threatening problems of BTHS. Aside from the Scientific and Medical Advisory Board of the BSF, there is no other forum where frank and important discussion about therapeutic ideas can meaningfully take place. Because BTHS individuals die each year from this disease, we need these conferences to communicate/discuss accomplishments, to stimulate new clinical and scientific progress (bench to bedside), and to encourage the scientific-medical community.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2014 ( Subtotal = $26,000 )
2014	2014	BARTH SYNDROME FOUNDATION INC	675 VFW PARKWAY	CHESTNUT HILL	MA	02467	MIDDLESEX	USA	Cardiovascular Diseases Research	000	1	6/24/2014	NEW	$13,000
2014	2014	BARTH SYNDROME FOUNDATION INC	675 VFW PARKWAY	CHESTNUT HILL	MA	02467	MIDDLESEX	USA	Cardiovascular Diseases Research	002	1	7/25/2014	NEW	$3,000
2014	2014	BARTH SYNDROME FOUNDATION INC	675 VFW PARKWAY	CHESTNUT HILL	MA	02467	MIDDLESEX	USA	Cardiovascular Diseases Research	001	1	6/26/2014	NEW	$10,000
														Subtotal = $26,000

Grand Total All Awards = $26,000

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Scientific and Medical Conference about Barth Syndrome

Award Number: R13HL124706

ORGANIZATION: NATIONAL HEART, LUNG, & BLOOD INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 06/25/2014

PERIOD OF PERFORMANCE END DATE: 05/31/2015

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