MDA advocacy in Washington
I have had the great privilege of witnessing some of the most transformative legislative moments in our MDA community’s history. While we have many heroes and legends among us, there are also many heroes whose names most will never know — people who, through their outreach to their elected officials, brought great champions to the cause of neuromuscular disease research and services.
The congressional funding bill spared government programs that sponsor neuromuscular research, but it doesn't protect them from upcoming deep sequester cuts
The news is generally favorable for many important government-funded neuromuscular disease research projects, in the wake of Congress’ recent passage of a continuing resolution funding the government through the end of September.
Most government operations, except those funded through the full-year appropriations bills, will be maintained at essentially the same level as fiscal year (FY)...
This international event seeks to foster cooperation and encourage research on behalf of people with rare diseases — and here's one way you can do just that
In the United States, a disease or disorder is defined as “rare” when it affects fewer than 200,000 Americans at any given time; in Europe, when it affects less than one in 2,000 people.
Clearly, we’re talking about very small numbers of people. But put them all together, and the numbers tell a different story. More than 6,000 rare diseases affect more than 60 million people in Europe and the U.S...
Since 2001, the MD CARE Act has had a tremendously positive impact on MD research — but its reauthorization this year will require active outreach
Note: The MD CARE Act is a critical part of the historic effort to find cures for muscular dystrophies. Watch for articles in the coming weeks that delve further into the remarkable progress attributable to this important legislation.
MDA asks Sarepta Therapeutics' CEO Chris Garabedian about next steps for this promising new drug for DMD
MDA is pushing forward on all levels to accelerate the pace at which we’re able to translate scientific discoveries into improved health outcomes for individuals living with muscle disease.
The SMA Project, a National Institutes of Health program supported by SMA patient advocacy groups, reports on its therapy development strategy in spinal muscular atrophy
A small-molecule drug candidate for spinal muscular atrophy (SMA) and an effective strategy for advancing therapy development are the primary outcomes being reported by the Spinal Muscular Atrophy (SMA) Project, a program established in 2003 by the National Institute of Neurological Disorders and Stroke (NINDS) to accelerate the development of therapeutic candidates for thi
MDA is partnering with federal agencies and other groups serving youth with disabilities to break down barriers for young people transitioning from childhood to adulthood
Like many high school seniors, Drew is busy choosing a college and preparing for the big transition from life at home to life on a college campus. Unlike his peers, Drew has Duchenne muscular dystrophy.