Current Actions

  • Call on your Congressmembers to attend the May Rare Disease Congressional Caucus Briefing!

    On May 18th, the Rare Disease Congressional Caucus will be hosting a briefing on "Incentivizing Innovation for Rare Disease Treatment Developmment." This event will brief congressional offices and their staff on the need for incentivizing innovation in treatment development and the value it provides to rare disease patients.

    You can find out more about the event by CLICKING HERE or reach out to the EveryLife Foundation (hclauson@everylifefoundation.org).

    Please take a minute to phone your legislators and urge them to send staff to the event! 

  • Urge Senator Kamala Harris to sponsor the OPEN ACT to repurpose rare disease treatments!

    Call on Senator Harris to be the lead sponsor of the OPEN ACT - Orphan Product Extensions Now, Accelerating Cures & Treatments (HR 1223).  The OPEN ACT could bring hundreds of safe, effective, and affordable medicines to rare disease patients within the next several years by incentivizing drug makers to repurpose therapies for the treatment of life-threatening rare diseases and pediatric cancers. The OPEN ACT could allow for insurance reimbursement of otherwise off-label treatments or procedures, and reduce the cost of orphan therapies. EveryLife Foundation, the National Organization for Rare Disorders (NORD), Global Genes, Genetic Alliance and an additional 192 patient organizations support this bipartisan legislation.

    For further information on the OPEN ACT, click here.

    TAKE ACTION: We are seeking members of CALIFORNIA'S ACADEMIC COMMUNITY to sign-on to the letter below!

  • Urge Senator Kamala Harris to sponsor the OPEN ACT to repurpose rare disease treatments!

    Call on Senator Harris to be the lead sponsor of the OPEN ACT - Orphan Product Extensions Now, Accelerating Cures & Treatments (HR 1223).  The OPEN ACT could bring hundreds of safe, effective, and affordable medicines to rare disease patients within the next several years by incentivizing drug makers to repurpose therapies for the treatment of life-threatening rare diseases and pediatric cancers. The OPEN ACT could allow for insurance reimbursement of otherwise off-label treatments or procedures, and reduce the cost of orphan therapies. EveryLife Foundation, the National Organization for Rare Disorders (NORD), Global Genes, Genetic Alliance and an additional 192 patient organizations support this bipartisan legislation.

    For further information on the OPEN ACT, click here.

    TAKE ACTION: We are seeking ORGANIZATIONS to sign-on to the letter below!

  • Call your Florida state legislators to co-sponsor Newborn Screening legislation!

    Take Action Today to Change Policy & Save Lives!  SB 1124 (Book) and HB 963 (Fitzenhagen) would provide a much needed, science-based approach to improving public health for all Florida’s children by expanding the state’s newborn screening process. This bill would save the lives and reduce the suffering of babies by allowing Florida to review and screen for all diseases that are recommended by a federal scientific committee, ensuring the earliest access to treatment. 

    Please take a minute to phone your legislators and urge them to co-sponsor SB 1124 and HB 963! 

  • Please ask Florida's Legislators to improve newborn screening

    Take Action Today to Change Policy & Save Lives!  SB 1124 (Book) and HB 963 (Fitzenhagen) would provide a much needed, science-based approach to improving the state’s newborn screening process. This bill would save the lives and reduce the suffering of babies by allowing Florida to review and screen for all diseases that are recommended by medical experts, ensuring Florida babies have the earliest access to treatment.

    The Florida State Senate will be hearing SB 1124 on Monday, March 27th.

    Please take a minute to email your legislators and share this action alert on social media! All you have to do is enter your address below to find your representatives, customize the letter if you wish and click submit.

  • Ask Your Representative to Co-Sponsor the OPEN ACT to Repurpose Drugs for Rare Disease Patients

    Take action to support the OPEN ACT - Orphan Product Extensions Now, Accelerating Cures & Treatments (HR 1223).  The OPEN ACT could bring hundreds of safe, effective, and affordable medicines to rare disease patients within the next several years by incentivizing drug makers to repurpose therapies for the treatment of life-threatening rare diseases and pediatric cancers. The OPEN ACT could allow for insurance reimbursement of otherwise off-label treatments or procedures, and reduce the cost of orphan therapies. EveryLife Foundation, the National Organization for Rare Disorders (NORD), Global Genes, Genetic Alliance as well as more than 220 patient organizations support this bipartisan legislation.

    For further information on the OPEN ACT, click here.

    Please take a minute to phone your legislators and share this action alert on social media! All you have to do is edit the letter below to advocate for your disease community, enter your name, address, and click submit.

  • Sign-On to Support Newborn Screening in Florida

      

    The EveryLife Foundation for Rare Diseases is proud to support SB 1124 (Book) and HB 963 (Fitzenhagen), which would improve the health of babies born in Florida by expanding newborn screening to include diseases recommended by the state's Genetics and Newborn Screening Advisory Council. Florida had been a leader in newborn screening, but its current approach to reviewing and approving screening for additional diseases has created a lag behind current medical recommendations. 

    Delays in diagnosis and treatment can cause severe cognitive and physical problems, and even death. Early treatment can prevent the irreversible complications of the disease, providing cost savings to the state and ensuring better health outcomes for babies born in Florida.

    TAKE ACTION: We are seeking ORGANIZATIONS to sign-on to the letter below!

  • Ask Your Legislators to Co-Sponsor the OPEN ACT to Repurpose Drugs for Rare Disease Patients

    Take action to support the OPEN ACT - Orphan Product Extensions Now, Accelerating Cures & Treatments (HR 1223).  The OPEN ACT could bring hundreds of safe, effective, and affordable medicines to rare disease patients within the next several years by incentivizing drug makers to repurpose therapies for the treatment of life-threatening rare diseases and pediatric cancers. The OPEN ACT could allow for insurance reimbursement of otherwise off-label treatments or procedures, and reduce the cost of orphan therapies. EveryLife Foundation, the National Organization for Rare Disorders (NORD), Global Genes, Genetic Alliance and an additional 175 patient organizations support this bipartisan legislation.

    For further information on the OPEN ACT, click here.

    Please take a minute to phone your legislators and share this action alert on social media! All you have to do is edit the letter below to advocate for your disease community, enter your name, address, and click submit.

  • Sign on to support an end to the NIH/FDA hiring freeze!

    President Trump instituted an immediate hiring freeze on January 23rd, signing a presidential memorandum that would affect a large swath of the executive branch including the National Institutes of Health (NIH) and the Food and Drug Administration (FDA). Unfortunately, this freeze will significantly hamper research into rare disease treatments and the subsequent approval process. We are seeking patient organizations to have our voice heard. 

    Sign your patient organization on to the letter below and lend your support to ensuring that the NIH and FDA have the resources they need to support rare disease patients!

  • Please tell your Missouri Representative to advance life-changing newborn screening for MPS II and SMA

    Dear Friend:

     

    We have 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).

     

    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. 

     

    Please take 3 minutes to ask your state Representative to support HB 66 by entering your information below!

     

    Working together, we can find solutions that turn hope into treatments.

     

    Thank you for your support and advocacy!

     

    Sincerely,

     

     

    Stephanie Bozarth, MSW

    National MPS Society

    Chairman, Board of Directors

    Chair, Advocacy Committee


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  • This week, call on your legislators to advance #CuresNOW!

     

    Take action to support the 21st Century Cures Act! This vital piece of legislation includes billions of dollars in new funding for critical research at the National Institutes of Health, new funding to accelerate drug approval at the Food and Drug Administration, and several provisions like the OPEN ACT and Priority Review Voucher Program that could lead to hundreds of new therapies for rare disease patients. We must unite and make sure that Congress gets our message loud and clear. This is our last chance to make Cures a reality. Call TODAY!

    Please take a minute to phone your legislators and share this action alert on social media!

  • Call on your Congressmembers to attend the September Rare Disease Caucus Briefing!

     

    On September 14th, the Rare Disease Congressional Caucus will be hosting a briefing on "Strengthening Medical Innovation in America for Rare Disease Patients!" This event will brief congressional offices and their staff on the need for innovation in biotechnology and the value it provides to rare disease patients. You can find out more about the event by CLICKING HERE.

    Please take a minute to phone your legislators and urge them to send staff to the event!

  • On June 7th, call on your Senators to advance #CuresNOW!

    On June 7th take action to support the Senate Cures Legislation! This vital piece of legislation includes billions of dollars in new funding for critical research at the National Institutes of Health, new funding to accelerate drug approval at the Food and Drug Administration, and several provisions that could be a game changer for the rare disease community. The House companion legislation 21st Century Cures Act passed the House, and in order to pass the Senate, we must unite and make sure the Senate gets our message loud and clear.

    Please take a minute to phone your legislators and share this action alert on social media!

  • Permission to Use Photograph

    Subject: 21st Century Cures Act / Senate Cures Legislation Action Alert

  • Ask your Legislator to Join the Rare Disease Caucus

    Help us strengthen the rare disease community's voice on Capitol Hill!  Please take 3 minutes to ask your legislator to join the Rare Disease Caucus.  It's easy - just enter your name and address & click send.  We also encourage you to personalize the letter to share information about your specific disease.

    If your legislator is already on the Caucus, the letter will automatically populate as a thank you letter instead - these are just as important to send!

    It can take up to 10 letters from constituents for a Member to respond so please share this Action Alert with your friends, family & colleagues. 

  • Call Your Legislator Today!

    As a signing organization in support of SB 1095 (California), we once again need your help to move this newborn screening bill off of the Senate Appropriations Committee suspense file. 

     Please call your legislator today to let her know why your organization supports SB 1095:

  • Sign on to support the OPEN ACT

     Please sign-on and urge Senators Murray and Cantwell to support the OPEN ACT below!

  • Ask Your California State Legislators to Support SB 1095 to ensure rare disease patients have the earliest access to treatments!

    Take Action Today to Change Policy & Save Lives!  SB 1095 would provide a much needed, science-based approach to improving public health for all California’s children by expanding the state’s newborn screening process. This bill would save the lives and reduce the suffering of babies by allowing California to screen for all diseases that are recommended by a federal scientific committee, ensuring the earliest access to treatment. The EveryLife Foundation for Rare Diseases, Muscular Dystrophy Association, Global Genes and 50 other organizations support this lifesaving legislation.

    Please take a minute to email your legislators and share this action alert on social media! All you have to do is edit the letter below to advocate for your disease community, enter your name, address, and click submit.

  • Sign on to support the OPEN ACT

    Please sign-on and urge Senator Warren to support the OPEN ACT below!

  • Sign-On to Support Newborn Screening

     

    The EveryLife Foundation for Rare Diseases is proud to sponsor and support SB 1095 (Pan), which would improve the health of babies born in California by expanding newborn screening to include diseases on the federal Recommended Uniform Screening Panel (RUSP). The Secretary of the United States Department of Health and Human Services, convenes a committee of newborn screening experts to develop the RUSP.  The federal process is rigorous, evidence-based, and science-driven, but ultimately states are not required to follow its recommendations.  This results in babies in some states receiving timely diagnosis and treatment, while babies born in other states do not, with potentially life-threatening consequences. 

    Because of the rarity of these diseases, it can take more than 7 years for a patient to receive a proper diagnosis.  Delays in diagnosis and treatment can cause severe cognitive and physical problems, and even death. Early treatment can prevent the irreversible complications of the disease, providing cost savings to the state and ensuring better health outcomes for babies born in California.

    Currently, California has to introduce new legislation every time a disease is added to the federal RUSP.  SB 1095 removes the legislative delay, so California can rapidly implement the new screen, ensuring babies in California have the earliest opportunity to receive lifesaving treatments. 

    TAKE ACTION: We are seeking ORGANIZATIONS to sign-on to the letter below!

  • Ask Your Legislators to Co-Sponsor the OPEN ACT to Repurpose Drugs for Rare Disease Patients

    Take action to support the OPEN ACT - Orphan Product Extensions Now, Accelerating Cures & Treatments (HR 971/S 1421).  The OPEN ACT could bring hundreds of safe, effective, and affordable medicines to rare disease patients within the next several years by incentivizing drug makers to repurpose therapies for the treatment of life-threatening rare diseases and pediatric cancers. EveryLife Foundation, the National Organization for Rare Disorders (NORD), Global Genes, Genetic Alliance and an additional 155 patient organizations support this bipartisan legislation.

    Please take a minute to phone your legislators and share this action alert on social media! All you have to do is edit the letter below to advocate for your disease community, enter your name, address, and click submit.

  • Ask Your Senators to Co-Sponsor the OPEN ACT to Repurpose Drugs for Rare Disease Patients

    Take action to support the OPEN ACT - Orphan Product Extensions Now, Accelerating Cures & Treatments (HR 971/S 1421).  The OPEN ACT could bring hundreds of safe, effective, and affordable medicines to rare disease patients within the next several years by incentivizing drug makers to “repurpose” therapies for the treatment of life-threatening rare diseases and pediatric cancers. EveryLife Foundation, NORD, Global Genes, Genetic Alliance and an additional 155 patient organizations support this bipartisan legislation. Please take 2 minutes to email your Representatives and share this action alert on social media!