Rare Disease Research Letter to the Honorable Kathleen Sebelius From Senator Franken and Governor Dayton

April 7, 2014
The Honorable Kathleen Sebelius
Secretary
U.S. Department of Health and Human Services 200 Independence Avenue, SW
Washington, DC 20201

Dear Madam Secretary:

As strong supporters of the NIH, we are contacting you on behalf of a constituent who participated in the National Institute on Aging Protocol 2003-086: "Clinical and Molecular Manifestations of Heritable Disorders of Connective Tissue." This individual received an abrupt notification that the NIA has discontinued the study prior to the expected conclusion date.
It is our understanding that this study was the only national research study focused on Fibromuscular Dysplasia and Ehlers-Danlos, Marfan, hereditary aneurysm, and Stickler syndromes. This type of research is incredibly important because it is the only source of information for doctors and patients on the causes, risk factors, and treatment options for these rare conditions. As you know, without the support of the NIH, rare diseases are unlikely to attract large amounts of research attention or funding.
We are concerned that the NIA's sudden discontinuation of the study was unnecessarily abrupt and caused undue hardship , especially given the importance of this study to the individuals and families who have committed time, energy and hope to the research process. The notification to study participants also did not provide a full and complete explanation as to why the study was discontinued. Therefore, we respectfully request a detailed explanation as to why the study was discontinued, as well as information on any plans to continue research on these rare diseases in the future.

Thank you for your attention to our request. 

Al Franken
United States Senator

Mark Dayton
Governor

CC: Francis Collins, Director, NIH 


It is not to late to sign the petition and leave a comment! https://www.change.org/petitions/nih-keep-hope-alive-and-restore-lifesaving-study

Wall Street Journal Article: http://online.wsj.com/news/articles/SB10001424052702304732804579425633803175144?mg=reno64-wsj&url=http%3A%2F%2Fonline.wsj.com%2Farticle%2FSB10001424052702304732804579425633803175144.html