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Ask Your Members of Congress to Join the Rare Disease Congressional Caucus

Help us strengthen the rare disease community’s voice on Capitol Hill! The Rare Disease Congressional Caucus is a forum for Members of Congress to voice constituent concerns, collaborate on ideas, facilitate conversations between the medical and patient community and build support for legislation that will improve the lives of people with rare diseases. Click here […]

Kids v Cancer is asking YOU to support the RACE for Children Act

The RACE for Children Act (Research to Accelerate Cures and Equity Act) would provide that companies developing a cancer drugs would undertake Pediatric Research Equity Act studies of their drug in children when the molecular target of their drug is relevant to a children’s cancer. To learn more and sign on in support, click here.

The EveryLife Foundation seeks your support to end the NIH/FDA hiring freeze

On January 23rd, President Trump instituted an immediate hiring freeze that would affect many federal agencies including the National Institutes of Health (NIH) and the Food and Drug Administration (FDA). Unfortunately, this freeze could significantly hamper research into rare disease treatments as well as the review and approval of new medicines. The EveryLife Foundation for […]

Join Research!America in calling for a standing fund to enable CDC/NIH response in public health emergencies

Earlier this week, National Institute of Allergy and Infectious Diseases (NIAID) Director Anthony Fauci, M.D. made the case for a standing fund to enable rapid Centers for Disease Control and Prevention (CDC) and National Institutes of Health (NIH) response in the event of public health emergencies. Research!America has issued an action alert to echo his call to […]

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 […]

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 […]

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 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!

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 […]