Newsletter: EveryLife Foundation Faster Track – October, 2018

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In Testimony, Foundation Board Chair Urges Senate to Act on Life-Saving Legislation

On Wednesday, October 3rd, Mark Dant testified before the Senate Health, Education, Labor and Pensions Committee’s Children and Families Subcommittee on the importance of expedited treatments for rare disease patients.  Mr. Dant urged Congress to pass the OPEN ACT (Orphan Product Extensions Now, Accelerating Cures and Treatments; S. 1509 and companion bill H.R. 1223) and to fund a Center of Excellence for Rare Diseases and more specialized review divisions at the Food and Drug Administration.  To read a copy of Mr. Dant’s full testimony, click here. To view the October 3rd briefing, click here.

Help us Advance Rare Disease Policy – Register Today for Our Community Congress Meeting

Join us on Wednesday, October 24th as we convene our annual in-person Community Congress meeting at the City Club of Washington. Held from 9:00am – 2:30pm, this policy discussion is the community’s chance to help influence the Foundation’s policy focus in 2019. The morning session, held from 9:00am – 12:30pm, is open to both members and prospective members. The afternoon session, held from 12:30pm – 2:30pm, is restricted to members only and will consist of an in-depth discussions on Newborn Screening, Regulatory, and Public Policy. Those interested in attending can register here. A draft agenda is available on the registration site.

Kick-Off the J.P. Morgan Healthcare Conference at A Rare Affair for Rare Diseases on January 6th

Are you or your colleagues attending the annual J.P. Morgan Healthcare Conference? If so, please join us at the famous Sir Francis Drake Hotel in Union Square for A Rare Affair. This spectacular networking and wine tasting event to benefit the EveryLife Foundation will be held the Sunday before the start of the conference on January 6th, 2019, from 6:30 pm until 10 pm. The event draws finance and biotech executives, venture capitalists, wine enthusiasts and rare disease advocates.  Until November 30th, the EveryLife Foundation is offering discounted “early bird” tickets to this exclusive event. To purchase tickets, click here. Tickets routinely sell out, so purchase a sponsorship to guarantee seating. For more information, please visit www.rareaffair.org or email Carol Kennedy at ckennedy@everylifefoundation.org.

Enter Your Art in the Rare Artist Contest by October 19th

Only one week remains for entries in the 10th annual Rare Artist contest. Don’t miss your chance at the more than $3,000 prize pool! This is a great opportunity for members of the rare disease community to share their talents with the world. The contest recognizes two entrants from each category (Children, Teen, Adult and Adult Digital and Photography): one by popular vote, and another chosen by a panel of leaders from the rare disease community. Entering is easy on the Rare Artist contest Facebook page.  Not an artist? Visit the Rare Artist gallery on Facebook to view the amazing entries and vote for your favorite in each category. Voting is allowed once every ten days and will remain open through October 26th. All members of the rare disease community – including patients, caregivers, family, researchers and medical professionals – are welcome to enter artwork. For more information on the contest, visit the Rare Artist website. For all additional questions, please contact the contest curator, Grant Kerber, at gkerber@everylifefoundation.org.

Apply Now for Rare Disease Week Travel Stipends and 2019 Event Sponsorships

Applications for Rare Disease Week on Capitol Hill 2019 travel stipends are now available. Two representatives from each state will be eligible for stipends to attend Rare Disease Week on Capitol Hill, with stipend amounts ranging from $200 to $1000 depending on distance from Washington, DC. For more information on Rare Disease Week on Capitol Hill 2019 and to apply, click here.  Applications for travel stipends for Rare Disease Week on Capitol Hill are open through December 14th.

Additionally, the Foundation’s Rare Giving program is now accepting applications for sponsorship of upcoming rare disease events that encourage collaboration among rare diseases and/or promote audience engagement in advocacy for public policy. Examples of events that we have financially supported include the Conference for Adrenal Insufficiency, Utah Rare Symposium, Family Education Summit at Camp Sunshine, PFDD meeting for Charcot-Marie-Tooth and Related Inherited Neuropathies. To request sponsorship of your 2019 event, please submit a letter of request that includes the projected number of attendees, participating organizations, diseases covered, agenda and website links along with sponsorship levels and benefits to lcundiff@everylifefoundation.org by November 30th, 2018.

RareHub Offers Innovative Solution for Patient Organizations in Washington, DC

As part of our commitment to cross-disease collaboration and legislative advocacy, the Foundation is pleased to introduce our Rare Hub program, which will provide a shared office space for rare disease organizations in Washington, DC. This office space will be a place to foster alliances and innovation among rare disease organizations and amplify the community’s voice on Capitol Hill. We are pleased to have Parent Project Muscular Dystrophy, PKD Foundation, Children’s Cause for Cancer and the National Fragile X Foundation as Partners in our Rare Hub.  For more information on Rare Hub, visit the program homepage. Save the Date for our Open House on Tuesday December 4th!

Want To Join Our Expanding Team? EveryLife is Hiring!

We’re bringing some new faces to the EveryLife family. Are you a driven team-player who’s passionate about rare disease advocacy? Then we want you on our team! We are seeking applicants for the following roles:

We request that all applicants be based in Washington, DC, or the surrounding area. For more information on EveryLife job opportunities, visit our careers page.

Looking to stay informed on Foundation activities?  Follow the EveryLife Foundation on Facebook and Twitter. If you would like to sign up for our monthly newsletters, click here.

Press Release: State Senator Richard Pan Introduces Lifesaving Legislation to Make California the Nation’s Leader in Newborn Screening

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Monday February 29, 2016, Novato, Calif. – On Rare Disease Day, the EveryLife Foundation for Rare Diseases applauds Senator Richard Pan, MD (D-Sacramento) for introducing SB 1095.  The Foundation is joined by the MLD Foundation, National MPS Society, Acid Maltase Deficiency Association (AMDA), the Ryan Foundation, Aidan Jack Seeger Foundation, and Jonah’s Just Begun Foundation to Cure Sanfilippo in supporting this lifesaving legislation to expand California’s newborn screening program.

Because of the rarity of these diseases, it can take more than seven years for a patient to receive an accurate 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.  California saves $9.32 in health care costs for every dollar spent on newborn screening.

“This bill will solidify California as the national leader in ensuring babies are screened for life-threatening rare diseases.  We are so grateful to Dr. Pan for his commitment to newborn screening. He is a hero to the rare disease community,” said Julia Jenkins, Executive Director of the EveryLife Foundation for Rare Diseases.

“Rare diseases often are difficult to diagnose in time before permanent damage is done,” said Dr. Richard Pan, a pediatrician and Senator representing the Sacramento region. “SB 1095 ensures California implements recommended newborn screening when an early diagnosis and treatment can prevent disability and save lives.”

The Secretary of the United States Department of Health and Human Services convenes a committee of newborn screening experts to develop a Recommended Uniform Screening Panel (RUSP).  The federal process is rigorous, evidence-based, and science-driven, but ultimately states are not required to follow the 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.

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.

The EveryLife Foundation for Rare Diseases is a 501c3 public charity dedicated to accelerating the pace of biomedical innovation through science-driven public policy.  www.EveryLifeFoundation.org

Press Release: Governor Jerry Brown Signs Legislation Ensuring Babies Born in California Receive the Earliest Treatment for Life-Threatening Conditions

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Friday, September 16, 2016, Novato, Calif. – On September 16th, Governor Jerry Brown signed into law Senate Bill 1095 (Pan), sponsored by the EveryLife Foundation for Rare Diseases. The bill ensures that babies born in California will be screened for a disease within two years of its addition to the federal Recommended Uniform Screening Panel.

Senator Richard Pan, MD (D-Sacramento) introduced the bill in February, noting that “[r]are diseases often are difficult to diagnose in time before permanent damage is done.” With leadership from the EveryLife Foundation and support from more than 120 patient organizations including March of Dimes, the National Organization for Rare Disorders (NORD), Muscular Dystrophy Association and Global Genes, this bipartisan legislation breezed through both chambers of the state legislature without a single “No” vote.

Every year, many California babies go undiagnosed for diseases and conditions that are easily detectable by newborn blood screening, a quick heel prick that all babies receive at the hospital shortly after being born. But while California was once a leader in newborn screening, the state does not currently screen for all of the diseases recommended by leading scientists and public health officials. Currently, new legislation must be introduced for the state to screen for any disease. As a result, California babies are delayed treatment, which can lead to unnecessary pain and suffering.

Sabrina Low-Dumond’s son Zack suffers from Pompe Disease. Because he was not diagnosed at birth, their Orange County-based family spent four anguishing years searching for answers and she notes that “[t]he delay caused by failure to screen at birth has left Zack with irreversible damage.”

She is not alone. Betsy Bombola of Napa lost her son Brian to MPS, or mucopolysaccharidoses, and remembers that “[t]he damage was irreversible and after 30 surgeries, including open heart, hip replacements, shunts, and corneal transplants, he died at just 34…”

With the Governor’s signature, the lives of babies born with MPS, Pompe, and many other diseases will be forever changed. They will get the earliest diagnosis and treatment possible for these life-threatening diseases. But SB 1095 doesn’t just save lives; it also saves money.  For every dollar spent on newborn screening, the state saves $9.32 in health care costs. The EveryLife Foundation for Rare Diseases applauds Senator Pan, Governor Brown, and the hundreds of advocates and patient organizations who helped make this possible.

 The EveryLife Foundation for Rare Diseases is a 501c3 public charity dedicated to accelerating the pace of biomedical innovation through science-driven public policy. www.EveryLifeFoundation.org

Newsletter: EveryLife Foundation Faster Track – June, 2017

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Newborn Screening Receives Funding Boost in Florida, Legislation Sent to Governor

Florida Governor Rick Scott has reached an agreement with state legislators on a budget for 2018, including provisions to implement screening for X-ALD and additional funding for Florida’s newborn screening centers, which have historically been significantly underfunded. This funding is crucial as it will allow the state to expand newborn screening for X-ALD and other diseases that could be screened for in future years. This is a major victory for the rare disease community, especially given that Governor Scott failed to increase funding for newborn screening in past budgets.

On June 5th, SB 1124, which would expand and enhance newborn screening in the state, was officially transmitted to Governor Scott from the legislature, where it passed with unanimous support in both chambers. The Governor has 15 days to act on the legislation.

The Foundation, in partnership with more than 100 patient organizations that endorsed the bill, is calling on advocates in Florida to contact Governor Scott to urge him to swiftly sign this critical legislation into law.

California Legislature Launches Bipartisan Rare Disease Caucus

Thanks to a partnership between the Foundation and California Action Link for Rare Diseases (CAL RARE), the California Legislature will launch a state caucus to tackle issues affecting California’s rare disease population.  The bipartisan Rare Disease California Caucus will be led by Assemblyman and Health Committee Member Rob Bonta (D-Oakland) and Assemblyman and Health Committee Vice Chair Brian Maienschein (R-San Diego).

In-District Lobby Days Provide Opportunity to Meet with Members of Congress

Rare Disease Legislative Advocates (RDLA) is hosting In-District Lobby Days to facilitate meetings for rare advocates across the country with their federal elected officials during the summer Congressional recess (August 7th through September 6th). Registration is open through July 4th. You can specify when you are available for meetings as well as the distance you are willing to travel.

Members of Congress need to hear regularly from constituents affected by rare disease, and meeting with them throughout the year is critical to building a relationship. These meetings are an opportunity to invite them to join the Rare Disease Congressional Caucus, and to highlight legislation that could be beneficial to the rare disease community as well as legislation that causes concern.

In order to help advocates prepare for effective meetings, RDLA will host a webinar on July 28th. Those registered for In-District Lobby Days will be invited to participate, and a recording will be available for anyone unable to join live. Brief background documents on legislation of greatest interest to the rare community will be available online next month as a resource.

Don’t miss this opportunity to let your Members of Congress know which federal issues are of greatest interest to you. Register today!

Registration Open for Scientific Workshop on Emerging Technologies for Rare Diseases

The Foundation will convene its ninth annual Rare Disease Scientific Workshop to explore emerging rare disease technologies on September 12th in Washington, DC. The goal of the workshop is to gather key thought-leaders from industry, the Food and Drug Administration (FDA), and patient organizations to discuss the latest biomedical advancements. Companies will present relevant case studies highlighting clinical and regulatory pathways to offer a deeper understanding of these emerging therapies. Senior leadership from the FDA will present an overview of resources and guidance available to patient organizations and industry. The conference is free to attend but participants must register in advance. Click here to register and view the latest agenda.

Ninth Annual Rare Artist Contest Now Open!

The Foundation is excited to announce that the ninth annual Rare Artist contest is now open for submissions.  This yearly contest, which celebrates the unique talents of the rare disease community, is open to rare disease patients, family members, caregivers and medical professionals.  The contest will accept entries through December 7th, and visitors to the Facebook gallery can vote for their favorites once every ten days until December 22nd.  Two awards will be given in each category: one to the entry receiving the most votes on Facebook, and the other selected by a panel of rare disease community leaders.  For more information on the Rare Artist contest, visit the program’s Facebook page.

If you plan to attend the Biotechnology Innovation Organization (BIO) International Convention in San Diego later this month, please visit one of the Rare Artist galleries that will be on display.  The Foundation will host a selection of works throughout the day during the Orphan and Rare Disease session track in room #3 on Tuesday, June 20th.  Additional works will be available to view during the BIO Exhibitor Hospitality Receptions taking place from 5:00 to 6:30 pm in the Exhibition Hall that evening.  We hope to see you there!

Nominations Now Open for Sixth Annual RareVoice Awards

Do you know a rare disease community leader who went above and beyond the call of duty in 2017?  Now is your opportunity to show appreciation by nominating them for a RareVoice Award!  Nominations are currently being accepted in the following categories: Congressional Staff, Government Agency Staff, State Patient Advocacy, Federal Patient Advocacy, and Teen Advocacy.  Nominations are open until July 31st, so be certain to submit your entry before it’s too late.

The RareVoice Awards will take place on Wednesday, November 15th, at Arena Stage in Washington, DC. For more information, visit the event website.

Thank you to our 2017 Sponsors, including Shire, Horizon, PhRMA, Vertex, BioMarin, Sanofi-Genzyme and Recordati.   Don’t miss your chance to show support for rare disease advocacy!  Contact Carol Kennedy, Chief Development Officer, at ckennedy@everylifefoundation.org for sponsorship opportunities.

Tickets Available for Italian Street Painting Marin on June 24-25th in San Rafael

Commemorating the 50th anniversary of California’s “Summer of Love”, the Italian Street Painting Marin festival in downtown San Rafael on June 24-25th will feature more than 100 madonnari (street painters) creating works influenced by this watershed moment in American history. Tickets are still available on Italian Street Painting Marin’s website.

If you attend, please visit the Foundation’s booth to meet our staff and learn about our latest initiatives.  Our booth will feature selections from the 2016 Rare Artist contest, accompanied by statements from the artists.  With the 2017 Rare Artist contest now open, this fun and family-friendly event could provide inspiration for your entry this year!

For more information on Italian Street Painting Marin, visit the event website.

Want to stay informed on Foundation activities?  Follow the EveryLife Foundation on Facebook and Twitter.  If you would like to sign up for our monthly newsletter, click here.