Here are some recent highlights from MDA's research program:

• MDA Awards 38 Research and Development Grants Totaling $12 Million
• MDA's Translational Research Program Funds Two New Corporate Drug Development Programs for DMD
• MDA's Clinical Conference
• MDA's New Symposium Series
• Funding Scientific & Clinical Conferences
• Save the Date!

The MDA Board of Directors approved funding for 38 research projects that were recommended by MDA's Medical and Scientific Advisory Committees (MAC and SAC). These grants became active on February 1, 2012.

• The new projects target 15 neuromuscular diseases in MDA's portfolio.
• These are in addition to 250 research projects currently funded by MDA.
• A third of the new projects focus on research in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).
• Six grants, totaling $2 million, were awarded for amyotrophic lateral sclerosis (ALS) research.
• Recent progress in fascioscapulohumeral dystrophy (FSHD) research was reflected in the funding of three new grants, bringing MDA's total current FSHD grant portfolio to $3.8 million.
• Other projects were funded to study mechanisms of stem-cell-mediated muscle regeneration, to improve the splicing defect of SMN2 and to improve muscle targeting of gene therapy vectors in spinal muscular atrophy (SMA); to characterize the role of dysferlin in limb-girdle muscular dystrophy (LGMD) type 2B; investigate siRNA-mediated suppression of the complement pathway in myasthenia gravis (MG); to investigate the use of antifibrotic strategies for the treatment of congenital muscular dystrophy (CMD); to conduct large-scale screening of small molecule therapeutics for myotonic dystrophy (DM1); and to devise an efficient method to reduce immunogenicity of enzyme replacement therapy in Pompe disease.
• See the Grants at a Glance feature for more about these new projects and to learn more about the researchers leading them.
• More information on the 300 active grants funded by the MDA

An important goal of MDA is to recruit and train the next generation of neuromuscular disease researchers. Six of the 38 new awards are Development Grants, awarded to senior postdoctoral fellows with the highest potential of developing into independent investigators. Recipients of development grants are mentored and trained by established neuromuscular disease researchers.

We are very thankful to the regular and ad hoc members of MDA's MAC and SAC for their expert reviews and funding recommendations.

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MDA's translational research program, MDA Venture Philanthropy (MVP), funds and facilitates the development of effective therapies for neuromuscular diseases. In December 2011, MDA's Board of Directors approved the funding of two new translational research projects recommended by MDA's Translational Research Advisory Committee (TRAC). Both grants, awarded to Tivorsan Pharmaceuticals ($1 million) and Summit plc ($750,000), aim to upregulate utrophin as a therapeutic strategy for DMD. These two grants will allow the development of two different classes of drugs as treatments for DMD. MDA's funding of Summit plc is to conduct a phase 1 clinical trial. Tivorsan's grant is to allow them to conduct all the required work-up necessary to obtain approval by the FDA to start a clinical trial.

We are very appreciative of the members of MDA's TRAC for their expert guidance, reviews and funding recommendations.

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MDA's Clinical Conference will be held in Las Vegas on March 4-7, 2012. The two-and-a-half-day program is focused on best practices and development of new therapies for neuromuscular diseases. Jerry R. Mendell, M.D., director of the MDA Clinic at Nationwide Children's Hospital in Columbus, and Michael E. Shy, M.D., co-director of the MDA Clinic at the University of Iowa, are serving as co-chairs for the conference. The conference will be attended by team members of MDA's approximately 200 clinics in the U.S., and a roster of the world's leading experts have been invited as speakers. All conference participants are encouraged to submit abstracts for consideration for poster presentations.

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MDA is launching a new symposium series. In 2012, there will be three symposia focused on topical issues relating to neuromuscular disease. Leading experts will meet to discuss and evaluate a specific issue, with the goal of addressing roadblocks in research progress, clinical management and the effective development of therapies for neuromuscular disease. Important conclusions will be published in appropriate scientific journals. The following three symposia are being planned for 2012:

• MDA Muscle Symposium: Newborn Screening in DMD
  Co-chairs: Jerry Mendell, M.D. & Michele Lloyd-Puryear, M.D., Ph.D.
• MDA Neuron Symposium: Role of Glia in Motor Neuron Degeneration in ALS
  Co-chairs: Stanley Appel, M.D. & Jeffrey Rothstein, M.D., Ph.D.
• MDA Translational Research Symposium: Enhancing Collaboration and Technology Transfer Between Academia and Industry
  Co-chairs: Elizabeth McNally, M.D., Ph.D. & Lee Sweeney, Ph.D.

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MDA is committed to enhancing the communication of new research findings and of information relating to the delivery of effective medical care for neuromuscular diseases. To achieve these goals, MDA awarded funds through its special grants program to help organize the following conferences:

• Congenital Muscular Dystrophy: Exploring the Role of the Myomatrix
• FASEB Summer Research Conference: Skeletal Muscle Satellite and Stem Cells
• Society for Muscle Biology / Frontiers in Myogenesis: Development, Function and Repair of the Muscle Cell

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Letters of Intent for the next round of Research Grant and Development Grant applications: June 15, 2012

MDA's Clinical Conference: Las Vegas, March 4-7, 2012 / Abstract submission deadline February 9, 2012

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