Here are some highlights from the summer (July-Sept. 2011):

• MDA Awards 40 Research and Development Grants Totaling $13.7 Million
• Funding Scientific & Clinical Conferences
• Evaluation of MDA's Translational Research Program
• Bridge-to-Industry Fellowship
• Cost of Illness Study
• MDA Telethon
• Featured Researcher
• Save the date!
• Announcements

In July 2011, the MDA Board of Directors approved funding for 40 research projects that were recommended by MDA's Medical and Scientific Advisory Committees (MAC and SAC).

• The new projects target 22 neuromuscular diseases in MDA's portfolio.
• These are in addition to some 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).
• Seven grants, totaling $2.5 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.
• Other projects were funded to study diabetes in Friedreich's ataxia; to improve cardiac and bone health in DMD; and to identify novel genes causing spinal muscular atrophy (SMA).
• See Grants at a Glance for more about these new projects.
• Click here for more information on the approximately 300 active grants funded by MDA.

An important mission of MDA is to recruit and train the next generation of neuromuscular disease researchers. Seven of the 40 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 diligent reviews and funding recommendations.

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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:

• ENMC Workshop on RyR1-Related Myopathies
• 8th International Myotonic Dystrophy Consortium Meeting
• ALS-MND Nursing Symposium 2011
• TREAT-NMD International Conference 2011
• Translational Research in Neuromuscular Diseases
• Limb-Girdle Muscular Dystrophy 2A Workshop
• RNA-Binding Proteins in Neurological Disease
• ALS Conference "Tarrytown House II"

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MDA's translational research program, MDA Venture Philanthropy (MVP), funds and facilitates the development of effective therapies for neuromuscular diseases. MDA has funded translational research since 2003, but the current MVP program, which has been in existence for the past three years, was comprehensively evaluated to assess its effectiveness and the content of its funding portfolio. Data were gathered from in-house records and an online survey of past and present grantees. MDA's Translational Research Advisory Committee (TRAC) met in August 2011 to evaluate the data and made several recommendations to improve the program. Some of these include the implementation of a training program for young investigators (see below); enhancement of the current process of financial and management diligence; and the recruitment of additional partners from a variety of fields.

Here are some achievements of the MVP program during the past three years:

• MDA funded 16 MVP projects, committing $10.3 million in grants.
• Most MVP projects are focused on pre-IND work-up of experimental therapeutics, although many also have included small phase 1 or 2a trials.
• All experimental therapies supported by MVP are continuing to progress through the drug development pipeline.
• Two-thirds of the companies funded by MDA obtained additional follow-on funding upon completion of the MDA-funded project.

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MDA is launching a pilot postdoctoral fellowship program called Bridge-to-Industry (B2I). The program is designed to train young research fellows in translational research through co-mentoring by established investigators in both industry and academia. The pilot program will be an integral component of MDA's translational research program and will launch in 2012.

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MDA has commissioned The Lewin Group to estimate the economic impact on families of ALS, various muscular dystrophies, myotonic dystrophy and spinal muscular atrophy. This will comprise medical and nonmedical costs, including costs associated with loss of productivity. There has not been such a comprehensive study done to date. Data analysis is ongoing, and we expect to release the results soon. We at MDA are grateful to all the family members who participated in the survey associated with this study.

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The 2011 MDA Labor Day Telethon raised more than $61 million, surpassing expectations. Broadcast nationally over six hours during prime time, the Telethon featured several research achievements by MDA grantees. MDA is grateful to the generous American public for its continued support of our mission.

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Carmen Bertoni, Ph.D.

Carmen Bertoni, Ph.D., an assistant professor of neurology at UCLA, has made a career in basic and translational research in Duchenne muscular dystrophy (DMD). She trained in Perugia (Italy) and at Tulane and Stanford universities in the U.S. prior to becoming an independent investigator at UCLA at the end of 2006. MDA identified Dr. Bertoni at an early stage and awarded her a Development Grant when she was a postdoctoral fellow in Tom Rando's lab at Stanford. She has received Research Grants from MDA for basic research in her lab, and is a recent recipient of an MVP grant from MDA to develop the next generation of "nonsense read-through" drugs for DMD. She has been reviewing grants for MDA since 2007 and was recently appointed to MDA's Scientific Advisory Committee.

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Letters of Intent for the next round of RG and DG applications: December 15, 2011. Read more about MDA Research Grants Programs

MDA Clinical Conference: Las Vegas, March 4-7, 2012

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Leon I. Charash, M.D.

Leon I. Charash, M.D., a pediatric neurologist who served as the chair of MDA's Medical Advisory Committee (1971-2008) passed away September 14, 2011.

"So many aspects of Muscular Dystrophy Association programs reflect his leadership and creativity. His strong commitment to research continues to help improve the lives of those living with neuromuscular diseases. We mourn the loss of a very dear friend, but we celebrate his many accomplishments," said R. Rodney Howell, M.D., Chairman of the MDA Board of Directors. Read more.

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