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NIH Report on Support for Research on Neuromuscular Diseases PowerPoint Presentation
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NIH Report on Support for Research on Neuromuscular Diseases
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  1. NIH Report on Support for Research on Neuromuscular Diseases Muscle Study Group Sept. 22, 2010 Glen Nuckolls, PhD Program Director for Muscle Diseases National Institute of Arthritis and Musculoskeletal and Skin Diseases National Institutes of Health US Dept. of Health and Human Services

  2. Topics for Presentation • NIH Funding for Neuromuscular Diseases • Examples of Recently Funded Grants • Funding Opportunities • Changes in Peer Review

  3. NIH Support for Neuromuscular Diseases (Dollars in millions and rounded)


  5. FY09 NIH Grants for the Muscular Dystrophies Training/career dev $2.2M Wellstone Centers $8.7M Multi-project grants $6.3M ARRA short-term grants $8.0M Translational grants $8.5M Small business $1.0M Investigator initiated single project grants $42.2M Other $5.9M $83M total

  6. Wellstone Centers Awards Pittsburgh Rochester Seattle Penn/Hopkins Iowa Children’s National (DC) North Carolina Rochester Boston Penn Iowa Nationwide Children’s (OH) 2003 2005 2007 2009 2011 2013 2015 NIAMS NINDS NICHD NHLBI

  7. Wellstone Center Competition 2010 The Research Institute at Nationwide Children’s Hospital Jerry Mendell, Chris Walker, Zarife Sahenk, Paul Martin Focus: Immune Response to Gene Therapy University of Pennsylvania Lee Sweeney, Elizabeth McNally, Melissa Spencer, Glenn Walter, Krista Vandenborne, Beth Barton, Michael Ostap, Richard Finkel Focus: Skeletal muscle and heart fibrosis in muscular dystrophies The University of Iowa Kevin Campbell, Steven Moore, Kathy Mathews, Carsten Bonnemann Focus: Dystroglycan-related muscular dystrophies


  9. Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy PI: Krista Vandenborne UF, Oregon HSU, CHOP, Penn Longitudinal study of 100 boys with DMD and 50 healthy controls Lower limb MRI and MRS to evaluate muscle structure and composition Measures of muscle strength and physical function Monitor participants as they begin standard corticosteroid treatment Banking of immortalized fibroblasts to facilitate subsequent genetic studies

  10. RC3: Pre-Clinical and Clinical Evaluation of Skeletal Muscle Activator, CK-2017357 for Myasthenia Gravis PI: Fady Malik CK-2017357 binds to the troponin complex and sensitizes muscle to nerve stimulation, increasing contractile force. Aims: Testing of pre-clinical efficacy in an AChR immune rat model Phase 2A clinical trial of single and multiple doses in MG patients

  11. Pre-Clinical Translational Research Exploratory/Developmental Projects in Translational Research for Neuromuscular Disease (R21) PAR-08-228 Cooperative Program in Translational Research for Neuromuscular Disease (U01) PAR-08-229 NIAMS Standard receipt dates through July 2011 Innovative Therapies and Tools for Screenable Disorders in Newborns (R03,R21,R01) PAR-10-230, PAR-10-231, PAR-10-232 NINDS NINDS SMA and DMD included Pre-clinical, clinical studies and clinical trials Standard receipt dates through Sept. 2013 NICHD

  12. Clinical Studies and Clinical Trials NIAMS Pilot and Feasibility Clinical Research Grants in Arthritis and Musculoskeletal and Skin Diseases (R21) PAR-10-282 NINDS Multiple receipt dates, next receipt Nov. 18, 2010 Clinical Studies of Safety and Effectiveness of Orphan Products Research Project Grant (R01) RFA FD-11-001 Two receipt dates: Feb. 2, 2011 and Feb. 1, 2012 NINDS Exploratory Clinical Trials (R01) NINDS Phase III Investigator-Initiated Multi-Site Clinical Trials (U01) PAR-10-198 & PAR-10-199 Standard receipt dates through March 2013

  13. Ancillary Studies NIAMS NINDS Ancillary Studies in Clinical Trials of CNS/PNS Disorders NINDS Accelerated Awards Program (R01) PAR-09-263 Multiple receipt dates, next date is Dec. 16, 2010 Ancillary Studies to Large Ongoing Clinical Projects (R01 & R21) RFAs AR-11-010 & AR-11-013 Letters of Intent Receipt Date(s): November 3, 2010; March 1, 2011   

  14. Peer Review Changes Phase out of A2 applications almost complete Guidance on making an application “new” Clarification of individual criterion scores Summary statement may report pre-discussion criterion scores Paragraph justification of impact/priority score Limitations on post-submission application materials (NOT-OD-10-115) Elimination of error correction window (NOT-OD-10-123)

  15. For questions and guidance about grant applications and other NIH issues: Glen Nuckolls NIAMS ( John Porter NINDS (