21st Century Cures Act HR 6


  • Your Representative


*Required fields

If you take action and have not already registered, you will receive periodic updates and communications from Parent Project Muscular Dystrophy.


Please vote YES on the 21st Century Cures Act (H.R. 6)

Dear [Decision Maker],

I am writing to you today to ask you to please vote YES on the 21st Century Cure Act (HR. 6).

The Duchenne community supports the efforts by Congress to speed the development of therapies for our community and thousands of others who desperately need them.

Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 4,000 live male births (about 20,000 new cases each year).

Duchenne results in progressive loss of strength and is 100% fatal patients typically only live until their mid twenties. There are no approved treatments for Duchenne and it is a race against time for all those diagnosed.

This bill contains provisions that will:

-Increase funding for the National Institutes of Health (NIH) & the Food & Drug Administration (FDA)
-Incorporate the patient perspective into the drug development and regulatory review process. (patient focused drug development)
-Clarify the use of platform technologies - Utilizing early data from trials to shorten follow on trials (such as in exon skipping)
-Provide new incentives for the development of drugs for rare diseases (OPEN Act)
-Invest in 21st century science and next generation investigators

I would also like to address my concern about the amendment offered by Rep. Dave Brat that endangers the enhanced NIH funding in the Cures Act. The Brat Amendment would undermine NIH's enhanced ability through H.R. 6, the 21st Century Cures Act, to perform basic research that contributes to the development of biomedical advances to the benefit of patients. We strongly urge you to oppose this amendment.

We hope you will keep our family in mind when you vote on this important legislation.

[Your Name]
[Your Address]
[City, State ZIP]
[Your Email]