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Cure CMD 2010 CMD Family Conference

Cure CMD 2010 CMD Family Conference. Mission. To raise CMD awareness and identify treatments for the CMDs

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Cure CMD 2010 CMD Family Conference

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  1. Cure CMD2010 CMD Family Conference

  2. Mission To raise CMD awareness and identify treatments for the CMDs The men who build the future are those who know that greater things are yet to come, and that they themselves will help bring them about. Their minds are illuminated by the blazing sun of hope. They never stop to doubt. They haven’t time. ~Micheal Egan

  3. Cure CMD Mission

  4. Cure CMD Goals: 2010-2011

  5. Goal 1: Discover CMD

  6. CMDIR Recently endorsed by AFM, Muscular Dystrophy Campaign (UK), Muscular Dystrophy Australia 23 countries represented Translated into: Portuguese, Spanish, German, French, English (Danish and Turkish forthcoming) Global registration= Power in Numbers and a Genetically Characterized Population

  7. CMD New Genes Ongoing international effort to identify new genes with a particular focus on: • Collagen VI like picture without collagen VI Mutation b. Dystroglycanopathy without 1/6 known gene mutations identified New Genes= New Targets

  8. CMD Diagnostic Guidelines CMD Standard of Care Committee, Brussels, Nov 2009 • CMD Care Guidelines • CMD Diagnostic Guideline Cure CMD is working with Dr. Sparks and Dr. Susana Quijano Roy to revise Genetest Diagnostic Guidelines= Recognition, Less Diagnostic Odyssey, Increased Rates of Genetic Confirmation of CMD community

  9. CMD Biomarkers A biomarker is “something” you find in the blood , urine or muscle or on imaging that helps you track or mark disease progression OR improvement • Biomarkers help a doctor monitor a disease • Biomarkers help a scientist tell if a drug is working • Biomarkers help confirm if a person who is treated says they feel stronger that there is a positive effect at the muscle level • Biomarkers can given information about rate of disease progression • Biomarkers can help a doctor titrate medication dose Biomarkers= Showing an Effect or Rate of Disease Progression on a Cellular or Organ System Level

  10. CMD Natural History Follow people with CMD over time, looking at biomarkers, outcome measures and imaging To lead to this: CMD Translational Grant: Jim Collins, Biomarkers, Jan 2010 CMD Quality of Life Survey, March 2010 ENMC CMD Outcome Measure Meeting, March 2010 CMD Comparative Outcome Measure study, June 2010 NINDS and CMD: Common Data Elements Meeting, Sept 2010 CMD Motor Outcome Measure Meeting, October 2010 Natural History Study=Validation of Outcome Measures, Increases Ability to Measure Drug Effect

  11. CMD Research • Partnered with SAM in 2009 to fund >200K in CMD Research • CMD Scientific Conferences: • July 2009 Therapeutic Targets in the CMDs • Jan 2010 CMD High Throughput Screening, NCGC Cure CMD SMAB, Sept 2010 Evaluate critical needs, direction and funding priorities Funding Research=Power to Identify and Evaluate Future Targets

  12. Goal 2: TREAT CMD

  13. Goal 3: Fundraise CMD

  14. Upcoming Events • Tully’s Run for Owen, August 21, 2010 • Cure CMD Team ~San Jose Rock N Roll ½ Marathon, October 3, 2010 • Rage Against the Disease Art Auction, October 10, 2010 • Cure CMD Team~Los Angeles Rock N Roll ½ Marathon, October 24, 2010 • Cure CMD Team ~Long Branch ½ Marathon, May 1st, 2011

  15. Stay on Mission, Build the Community, Prepare for clinical trials

  16. Manage a Roadmap Preclinical Trials Clinical Trials What target? What endpoints? What delivery system? How big is the effect? What preliminary data? Combination therapy? What validation workflow?

  17. CMD Target Roadmap

  18. Clinical Trial Readiness

  19. Clinical Trials Holy grail is not clinical trials, it is a treatment that works. To objectively determine a treatment works, we go through clinical trials. Right drug, right population. We cannot afford a major learning experience at the clinical trial level.

  20. Treatments • Clinical Trial Readiness • Collaborations • Roadmap

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