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Modular Ultrasonic Beacon System (MUBS) for Ultrasound-guided Interventional Surg

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 1R43AR065314-01
Agency Tracking Number: R43AR065314
Amount: $244,585.00
Phase: Phase I
Program: SBIR
Solicitation Topic Code: NIAMS
Solicitation Number: PA12-088
Solicitation Year: 2013
Award Year: 2013
Award Start Date (Proposal Award Date): N/A
Award End Date (Contract End Date): N/A
Small Business Information
3510 West Liberty Road
ANN ARBOR, MI 48103-
United States
DUNS: 829566004
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 (734) 369-4268
Business Contact
Phone: (734) 369-4268
Research Institution

DESCRIPTION: Myotonic dystrophy 1 (DM1) is the leading form of adult muscular dystrophy resulting in progressive neuromuscular effects. The disease affects 1 in 6000 people. Nymirum is interested in identifying a small molecule that can be used to slow oreliminate the progression of DM1. The cause of DM1 is the expansion of a CUG RNA sequence within the DMPK gene. This repeat RNA is toxic to normal cellular function by sequestering and altering the activity of needed splicing factors for normal neuromuscular function. We are taking an approach to identify small molecules that will bind to the expanded CUG RNA repeat to prevent sequestration of splicing factors and repair the abnormal splicing phonotype in cells. To this end we have identified 2 chemicalseries of small molecules with clear structure activity relationships for CUG RNA binding and rescue of abnormal splicing in a DM1 cell model. In this proposal, we aim to optimize the structural features of the series to find more potent binders and splicing rescue molecules. To do this we will utilize our extensive knowledge and tools related to RNA structure and small molecule interactions and bring it to bear on targeted medicinal chemistry. DM1 represents a major unmet medical need and patients are inneed of new drugs to slow or eliminate the neuromuscular deterioration PUBLIC HEALTH RELEVANCE PUBLIC HEALTH RELEVANCE: Myotonic dystrophy 1 (DM1) is a progressive neuromuscular disease in adults with no known cure. Nymirum has identified molecules with biological activity to alleviate the disease and will be optimizing them for better activity and drg development purposes.

* Information listed above is at the time of submission. *

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