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MDA's 2015 Scientific Conference points the way toward tomorrow's therapies

by Quest Staff on April 21, 2015 - 9:07am

Quest Spring 2015

Last month, MDA brought together more than 400 of the nation’s leading scientists, researchers and professionals specializing in neuromuscular disease research and care at its 2015 Scientific Conference in Washington, D.C., March 11–14. This pre-eminent gathering, hosted every other year, was designed to accelerate discoveries and drug development for families fighting neuromuscular diseases.

“One key strategy to accelerating lifesaving and life-enhancing treatments and cures for neuromuscular and motor neuron diseases is to ensure collaboration among the nation’s top leaders in our scientific community,” said MDA President and CEO Steven M. Derks. “I’m proud that, in addition to the hundreds of different research projects we fund, MDA continues to be a facilitator for researchers nationwide to share ideas, best practices and novel strategies through our scientific and clinical conferences.”

Translating hope into action

This year’s conference focused on better understanding disease causes, identifying potential therapies and discussing new advances in laboratory and clinical research.

Clinicians and scientists who attended were excited about the new developments in disease research and treatment they can take back to their labs and families they serve. MDA anticipates the number of promising human clinical trials will double in the next five years.

Here are some highlights of developments and important topics discussed at the conference, many of which may have spillover implications for multiple disorders:

  • Laboratory results demonstrate great promise for delivering stem cells and genes to muscles throughout the body.  
  • More research investment is needed to identify serum biomarkers of neuromuscular diseases, which are used to monitor disease progression and therapy effectiveness and are critical tools for clinical trials.
  • With the discovery of a new gene flaw linked to ALS (amyotrophic lateral sclerosis), researchers are quickly developing improved models that are essential for gaining a better understanding of what causes ALS and how to develop therapies.
  • Researchers are excited about the variety of different therapies that are in development to treat Duchenne muscular dystrophy (DMD). Someday, this will allow doctors to treat all people with DMD (regardless of their particular mutation) and to combine therapies for maximum effect.
  • Researchers see promise in the gene replacement therapies that are moving into clinical trials for muscular dystrophies. If these therapies are successful, similar strategies could be applied to a variety of neuromuscular diseases.
  • Researchers feel confident about the ability of new genetic technologies to pinpoint the underlying cause of disease in various congenital myopathies.
  • Researchers have made substantial progress in developing better models to study motor neuron diseases, including spinal muscular atrophy (SMA).
  • An encouraging number of therapies are currently being tested for SMA, including gene transfer therapy and gene-modifying strategies.

Read the daily conference updates to learn more about the specific research presented and for more details on the studies discussed.

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