Spring is a time of change and new growth, and MDA is experiencing a lot of both these days. No matter where you look, we’re innovating, extending our reach, leveraging our resources, supporting new initiatives and building partnerships.
In research, MDA is investing in drug development and working toward ensuring a clear pathway to approval for those experimental therapies that prove effective. Today, more clinical trials of experimental therapies for neuromuscular diseases are under way than at any point in history. Pharmaceutical companies are partnering to bring drugs to market. A coordinated public-private research infrastructure is beginning to emerge to support and speed the arduous process of determining if a drug that works in a mouse is safe and effective in a person. MDA is leading initiatives to routinely screen for some neuromuscular diseases at birth, in order to use existing therapies to prevent their onset.
MDA is mounting a large-scale effort to ensure the reauthorization of the MD CARE Act in Congress — legislation that has funded critical research infrastructure and improved health outcomes and clinical care interventions for people living with the various forms of muscular dystrophy. MDA also is working with other patient organizations to streamline the U.S. Food and Drug Administration’s approval process for amyotrophic lateral sclerosis clinical trials and drug development. Similarly, MDA is supporting passage of the ABLE Act, which provides an important financial tool for everyone in our MDA community. To learn how you can help in these and other efforts, see The Power of Building Relationships.
In addition to our existing clinical research networks for ALS and Duchenne muscular dystrophy, MDA just launched a third network for myotonic muscular dystrophy (MMD). Thanks to exciting MMD research advances, clinical trials are likely to begin soon in this disease and MDA stands ready to help make them happen. For more, see Research Updates Spring 2013.
Above all, spring is a time when we set a course for our future success, whether by planting seeds or nurturing those just beginning to grow. With your help, MDA is here to ensure that the exciting growth of neuromuscular research and care remains steady and strong.
Steven M. Derks
President and CEO