Dr. H. Orhan Akman: RGI BeHeard Grant Winner
The Rare Genomics Institute (RGI) is a non-profit organization that helps leverage the resources of industry and academic partners to promote research discoveries related to rare diseases. Its division, BeHeard, (Helping Empower & Accelerate Research Discoveries) offers premium biotechnology prizes to rare disease researchers.
Dr. H. Orhan Akman, Associate Research Scientist at Columbia University Medical Center in New York, has won a grant from the 2015 BeHeard Challenge. Dr. Akman’s grant is related to the mouse models he develops for APBD research. Dr. Akman, your work means so much to the APBD community. Congratulations!
Frequent misdiagnosis of adult polyglucosan body disease. – PubMed – NCBI
Hellmann MA, et al. J Neurol. 2015.
House overwhelmingly passes 21st Century Cures Act – Modern Healthcare
21st Century Cures (H.R. 6) bill passes in House by vote of 344-77Washington, D.C. – July 10, 2015 – The following statement was issued by Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD), on today’s approval by the House of the 21st Century Cures legislative initiative.
Today’s passage in the House of the 21st Century Cures (H.R. 6) bill is a huge win for the rare disease community, which actively supported the legislation. By promising mandatory funding for the NIH, strengthening the FDA’s ability to streamline the clinical trials process, and further incentivizing the development of orphan products, 21st Century Cures will help pave the way for developing new cures and treatments, thereby closing the gap for the vast majority of the 7,000 rare diseases that currently have no approved treatment. The legislation positions the U.S. to be the continued international leader in medical product innovation.
NORD congratulates the House, Energy & Commerce Committee and the other groups who worked to help this initiative pass. Today’s vote underscores the bipartisan commitment to move this legislation through the House and we hope the same spirit of collaboration leads to prompt development of similar legislation for consideration by the Senate.
Peter L. Saltonstall
President and CEO, National Organization for Rare Disorders (NORD)
May 17th, 2015
Congratulations to Dr. Heather Lau APBDRF Scientific Advisory Board member for being awarded a NTSAD grant.
Co-funder: The Canavan Foundation
* To define and categorize clinical endpoints that may be used for the development of clinical therapeutic trials.