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2015 Research Grant Opportunity Made possible by Team NTSAD, National Tay-Sachs & Allied Diseases Association (NTSAD), and the Orphan Disease Center at PennMedicine The 2015 Million Dollar Bike Ride Pilot Grant Program is now open. One $43,000 pilot grant is available focusing on forms of Tay-Sachs, Sandhoff, GM-1, or Canavan disease. We are soliciting proposals for innovative research projects that involve basic research, translational studies or clinical studies relevant to the diseases mentioned above. Projects may be focused on (1) technology approaches such as stem cells, molecular chaperones, substrate inhibitors, small molecule drug screening, gene therapy, novel drug delivery to the brain or on (2) other pre-clinical and clinical research needs, such as clinical outcome measures, registries, animal models, or biomarkers. This grant is made possible by Team NTSAD, the National Tay-Sachs & Allied Diseases Association, and the Orphan Disease Center at PennMedicine. Please first review the RFA guidelines before submitting your pre-application using the web form below. All pre-application submissions are due Monday, September 14, 2015 by 5:00 pm (EST). If your pre-application is approved, you will be notified with an invitation to submit a full-application, due October 19, 2015. For questions regarding this pilot grant program, please contact Samantha Charleston at This email address is being protected from spambots. You need JavaScript enabled to view it., or (215) 573-6822. Click here to view the 2015 MDBR Pilot Grant RFA Guidelines: 2015 MDBR Pilot Grant RFA Guidelines Click here to submit your pre-application: 2015 MDBR Pre-Application
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Last year's Million Dollar Bike Ride Grant recipient was Dr. Annette Bley, a pediatrician from Germany. She was awarded a grant to study the natural history of Canavan disease (CD). This work will allow Dr. Bley and others to learn more about the natural course of CD and to develop quantifiable measures of the disease.
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NTSAD is pleased to announce the 2015 Research Initiative Grants totaling $266,000 thanks to many generous research supporters.
This year NTSAD solicited proposals for innovative research projects that involve
Grant awards were based on final proposal evaluations by a Research Evaluation Subcommittee of NTSAD's Scientific Advisory Committee and other scientists who generously volunteered their time. Grants were made for a one to two year period for up to $40,000 per year. (Funding for a second year is predicated by adequate progress during year one.
View the full Request For Proposal
View Press Release
NTSAD would like to thank all of those that submitted an application this year. The pool of applicants was highly competitive and numerous proposals were conducive to the NTSAD mission. At this time NTSAD congratulates the five awardees for the 2015 research grants and includes their summaries below.
Project: Development and validation of a rapid, MS/MS-based method
to detect Hexosaminidase deficiency in Tay-Sachs disease
Project: Intravascular gene therapy for feline GM2 gangliosidosis
Project: Defining the Natural History of Canavan Disease through Development of an International Registry
The following two grants are made possible by the Katie & Allie Buryk Research Fund of NTSAD:
Project: Registry and Repository for Late Onset GM2 Gangliosidoses
Project: Generation of a knock-in mutant Hexb mouse model
For a description of each project's goals and impact, read more at 2015 Research Initiative Grants
NTSAD has maintained a strong commitment to funding research over the years to advance its mission to lead the fight to treat and cure Tay-Sachs, GM-1, Sandhoff, Canavan, and other related genetic diseases. Read about the NTSAD Research Initiative including highlights and past awards.
On May 7, 2015 the New York Times Well blog, Think Like a Doctor, presents the symptoms reported by twin sisters, Katie and Allie Buryk. Readers are asked to figure out the diagnosis and comment. The patient history, neurological notes and blood test results are offered as clues. Read the blog entry here: http://well.blogs.nytimes.com/2015/05/07/think-like-a-doctor-taking-a-stand/?ref=health&_r=0
Almost 300 responses were made. The New York Times posted on May 8 the diagnosis being Late Onset Tay Sachs and describes how the diagnosis was made. Less than a handful of the reader's comments mentioned Late Onset Tay Sachs. Read the blog diagnosis entry here: http://well.blogs.nytimes.com//2015/05/08/think-like-a-doctor-taking-a-stand-solved/
Articles published in the News Blog will have the headline appear on the web site's home page and also be listed on the News landing page with "read more".