As an advocate for patients with rare diseases you are a very important part of the legislative process. You can make the difference as you are the voices your legislators and congressmen want, or in some cases do not want to hear. Please complete the form below to take action and contact your Member of Congress

After you take action your job is not done! The final step is to share the action alert with your family, friends, co-workers and any other people that might be interested in taking action on behalf of the rare disease community.

National MPS Society urges your support in Missouri to advance life-changing newborn screening for MPS II and SMA!

The National MPS Society has a great opportunity for Missouri to include two genetic and potentially deadly diseases in their Newborn Screening Panel.  In the next week, the Missouri House of Representatives will be voting on HB 66 to expand the newborn screening requirements to include Hunter Syndrome (MPS II) and spinal muscular atrophy (SMA).

Missouri residents: Please take 3 minutes to ask your state Representative to support HB 66 by clicking here.

Newborn screening is necessary for MPS II and SMA because timely treatment allows for much better outcomes.  Without newborn screening it can take months or years to get a diagnosis.  During that time, the disease is causing irreversible damage throughout the body.  Currently, MPS II and SMA have treatments approved by the Food and Drug Administration with several more treatment options in the pipeline.  Newborn screening would ensure timely treatment of MPS II and SMA, which requires early intervention for the greatest benefit.


The American Academy of Pediatrics (AAP) needs you to voice your support for the safety of vaccines!

The American Academy of Pediatrics (AAP) is circulating a letter to President Trump expressing unequivocal support for the safety of vaccines.  The letter affirms the overwhelming scientific consensus that vaccines are safe, effective, and save lives.  The letter will be accompanied by a non-exhaustive listing of vaccine scientific evidence.  The letter is open to state and national organizations.  To add your organization, please complete the following form.  The deadline to sign on is COB Friday, February 3.  Should you have any questions, please contact Pat Johnson at

Submit your patient story to be hand-delivered to your Members of Congress!

Any rare disease patient or caregiver unable to participate in Rare Disease Week on Capitol Hill is encouraged to submit a personal story and photo to be hand-delivered to Congress on Lobby Day on March 1st. It is critical for each Member of Congress to hear from local individuals and families about the impact of rare disease and what public policy changes are needed to improve the lives of rare disease patients. Help us give the rare disease community a voice on Capitol Hill!


Rare Stories

  • Please list the website of your patient group or your personal website.
  • Please write a statement about how rare disease has affected your life and what you would like your U.S. Representative and Senators to do on behalf of the rare disease community.

Thank you for sharing your unique story with us as part of Rare Disease Week on Capitol Hill. The deadline for submissions is February 19, 2017.

Subscribe to RDLA’s Mailing List for the Latest Updates

As we head into a crucial juncture for the development of rare disease treatments, our advocacy efforts will play a central role ensuring the progress made by our community continues.  To receive the latest updates at the state and federal levels, sign up for the EveryLife Foundation and RDLA mailing list, and follow RDLA on Facebook and Twitter.  By taking these steps, you’ll be ready when our community needs to act.

Sign-On to Support the OPEN ACT

The EveryLife Foundation for Rare Diseases is asking patient organizations to sign-on in support of the OPEN ACT, legislation that could help double the number of treatments for rare diseases. Already, 173 national patient organizations have signed-on to support this life-saving legislation. Click here to add your organization’s name to the list. For additional background on the OPEN ACT, and to see a list of the signing organizations click here.

National Health Council Urges Patient Organizations to Show Support for 21st Century Cures

The National Health Council is circulating a sign-on letter to Congressional leadership to urge passage of the 21st Century Cures Act by the end of 2016. It is imperative that the tireless work by Congress and the patient community does not go to waste by delaying action until the next Congress in 2017.

For to view the letter and sign, please click here. The goal is to present the letter to House and Senate leaders before Congress comes back on Monday, November 14th, so signatures are requested no later than Thursday, November 10th.

Only the name of organizations will be listed on the letter, not the individual signers.  Please note that you must be authorized on behalf of your organization to commit to signing publicly.

If you have any questions about the letter, please contact Eric Gascho, Vice President of Government Affairs at the National Health Council, at or 202-973-0545.

Sign your Organization to Support the Rare Pediatric Disease Priority Review Voucher Program in 21st Century Cures!

The National Organization for Rare Diseases (NORD) has drafted a sign-on letter for organizations to show their support in reauthorizing the Rare Pediatric Disease Priority Review Voucher program as part of 21st Century Cures. NORD will distribute this letter throughout Congress in mid-November, so they urge your support in the next week.

CLICK HERE to sign your organization onto this letter today!

Energy and Commerce Committee Chairman’s Open Letter to Advocates on #CuresNow

Rare disease advocates across the country, including Emily Muller and Sharon Rose Nissley of Illinois as well as Shira Strongin of California, are helping to generate support for 21st Century Cures. And their efforts, as well as those of advocates meeting with Members of Congress and their staff in In-District Lobby Days meetings, are making a difference!

As U.S. House of Representatives Energy and Commerce Committee Chairman Fred Upton wrote in a recent editorial:

“Thanks to a remarkable outpouring and mobilization of rare disease advocates, August 2016 will go down in the books as the “Summer of Cures”… Together, we’ve made incredible strides in our effort to deliver #CuresNow. Every story, every single voice matters, and we are grateful for your support and willingness to share your personal experiences with disease.”

But time is running out for the Senate to act. As Chairman Upton noted, “It’s been a tremendous effort by the rare disease community and I am proud to report that we are closing in on the finish line. But we can’t get there without everyone’s continued efforts — now is the time to double down on all our efforts.

This month, the EveryLife Foundation for Rare Diseases is asking patients and advocates to submit photos and hashtag them with #CuresNOW. Lives are depending on it.

Take Part in the Rally for Medical Research!

Held every September, this Capitol Hill Day event continues the momentum established in 2013, and includes nearly 300 national organizations coming together in support of the Rally for Medical Research. Led by the American Association for Cancer Research, the purpose of the Rally is to call on our nation’s policymakers to make funding for National Institutes of Health (NIH) a national priority and raise awareness about the importance of continued investment in medical research that leads to MORE PROGRESS, MORE HOPE and MORE LIVES SAVED.

Click here to get learn more and get registered today!

Ask Congress to Fund More Childhood Cancer Research

When Congress returns from their August recess, they will be considering how much funding to provide the National Institutes of Health — and the National Cancer Institute for 2017. As reported on the Children’s Cause Cancer Advocacy blog, a House Appropriations committee recently approved a spending bill that would increase NIH funding by $1.25 billion, while Senate appropriators would increase the NIH budget by $2 billion.

Children’s Cause Cancer Advocacy is urging advocates to write to their Senators and Representatives and ask them to support the higher Senate number, so that the NIH and NCI continue to work for new treatments and explore the promise of immunotherapy.

Take action now and tell Congress why each additional research dollar matters for children with cancer.

Then, consider scheduling a district office meeting during this August recess. Face-to-face meetings with your elected officials and their staff in the district office are an extremely effective way to get to know them and express your views on key issues like this one. CCCA’s new step-by-step Guide to Congressional District Meetings can help you!