Helping families find the critical information and support they need
December 11, 2019
Report from Rare Disease Week in D.C.
March 2, 2017
2019 - The Year in Review
December 31, 2019
Help Protect All People with Rare Diseases
March 7, 2017
We are stronger together – over 30 million Americans are affected by a rare disease – 50% of them children! 80% are genetic. And less than 5% have effective treatments.
The National Institutes of Health (NIH) underscores the promise of research and the critical role patient organization need to continue playing in advocating for research to bring answers to our children.
There is universal acknowledgement that active advocacy and involvement by affected families was one of the most vital factors behind the successful passage of the 21st Century Cures Act last December. That historic legislation holds real promise to accelerate research and innovation in the treatment of rare diseases.
But much of the earlier progress could be dramatically reversed by the current Congress. Regardless of your personal politics, it is a fact that public policies made by the US Congress can either help or impede progress in research and new drug development. And proposals being debated to replace the Affordable Care Act present a major risk of a return to high risk pools for our children with exorbitant premiums, limited coverage, annual and lifetime caps, and even denial of any coverage. To learn more about Wishes for Elliott's involvement with Rare Disease Week, check out our blog! Click here to learn Seven Ways to Support SCN8A Children and Families.