Our Diseases May Be 'Rare,' but Our Voices Are Strong

Article Highlights:
  • Each of the more than 40 neuromuscular diseases in MDA's program is considered "rare;" however, when all those with rare diseases combine their efforts, they can be a mighty force for change.
  • In the coming months, MDA’s advocacy program will help unite the voices of those with rare diseases by highlighting opportunities such as outreach to elected officials and the upcoming MDA national neuromuscular disease registry.
by Annie Kennedy on March 31, 2011 - 9:01am

QUEST Vol. 18, No. 2

Did you know that each of the more than 40 neuromuscular disorders covered by MDA is considered a “rare disease” because each affects fewer than 200,000 people in the United States?

Rare diseases are at a disadvantage when it comes to therapy development, which requires decades of research, hundreds of millions of dollars of funding, and significant investment risk. It also requires an infrastructure that provides easy access to clinical trial participants, and a scientific way to judge a therapy’s effectiveness (outcome measures).

To support rare disease research, the federal government has enacted several policies over the last two decades, including the Orphan Drug Act and the establishment of the National Institutes of Health (NIH) Office of Rare Disease Research. Yet only 5 percent of rare diseases have seen any therapeutic benefit.

MDA has expedited therapy development for neuromuscular diseases by building innovative clinical research infrastructures, fostering partnerships with pharmaceutical companies and funding the establishment of outcome measures.

MDA’s translational research program has accelerated drug development; of the 14 projects funded by the program since 2004, 12 are still ongoing. In addition, many projects originally funded by MDA have attracted interest from companies that are paying for continued development.

However, there still is a long way to go — and your help is needed. In the coming months, MDA’s advocacy program will be highlighting opportunities to participate in infrastructure-building and information-gathering efforts, including: “disease impact” surveys and the online MDA clinic feedback survey; outreach to elected officials regarding the importance of the congressional Rare and Neglected Disease Caucus, and of federal funding for research; advocacy to reauthorize and update legislation creating research incentives; ongoing opportunities to participate in clinical trials; and the much-anticipated MDA national neuromuscular disease registry, which will be released through MDA clinics.

While each disease covered by MDA may be “rare,” combined we are a mighty force. There is great strength in our MDA community!

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