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Featured News
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Coming off of a thrilling Rare Disease Day and looking ahead to our summer events, there is a lot to be excited about in this month's newsletter. Read on for info about your state's grade in our new State Report Card and tickets to our West Coast bound Living Rare, Living Stronger NORD Patient & Family Forum — and so much more!
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NORD's Annual State Report Card Reveals Trends and Shortcomings in Rare Disease Healthcare Coverage
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The National Organization
for Rare Disorders (NORD) recently released our 2024 State Report Card, which grades all 50 U.S. states on critical issues impacting access to care for the 1 in 10 Americans living with a rare disease.
NORD’s State Report Card is a resource for patients, advocates, and policymakers to better understand how their state rates in areas of greatest need so they can take action at the local and state level. The report card includes newborn screening, Medicaid coverage, step therapy policies, telehealth, prescription drug out-of-pocket cost protections, and more.
Learn how your state measures up. Read the NORD State Report Card.
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Register for the Living Rare, Living Stronger Patient & Family Forum
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Registration for the 2024 Living Rare, Living Stronger NORD
Patient & Family Forum is now open! Join us on June 7-8, live from Universal Studios in Los Angeles, California and virtually, to hear real stories from our rare disease community and gain practical knowledge on how to and live your best rare life. We can't wait to host you there.
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Get Tickets to the Rare Impact Awards
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Following the Living Rare Forum, on June 8, NORD will be celebrating the 2024 Rare Impact Awards! This annual ceremony honors individuals, organizations, and companies who made invaluable achievements and advancements on behalf of the rare disease community. We will announce each of our honorees in the coming days — you don't want to miss this event!
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Submit Your Art to the Living Rare Forum Swag Design Contest
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NORD is providing an
exciting opportunity to showcase the artistic talents of our community as part the Living Rare, Living Stronger NORD Patient and Family Forum on
June 7-8 in Los Angeles, CA!
We invite individuals impacted by rare diseases to channel their creativity and share their unique interpretations of the event’s theme, “Lights, Camera, Rare Action.” The winning submission will be prominently featured on the official tote bag provided to all in-person attendees. The winner will also receive travel and lodging assistance for them and one guest to attend the event.
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Read through the contest rules, eligibility requirements, and full submission guidelines here.
Only submissions received by 11:59 pm EST on March 29, 2024, will be considered.
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NORD Breakthrough Summit Poster Abstracts
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We are looking for poster abstracts featuring innovative rare disease research to display at the NORD Rare Diseases + Orphan Products Breakthrough Summit in DC this October. The Poster Hall features research on topics such as advancing patient care, addressing health disparities, and increasing patient advocacy and engagement. This is a great opportunity to put your research in front of a large audience. View the full guidelines here.
The deadline to submit is Wednesday, June 12, 2024
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Attention Corporate Patient Assistance Leaders:
Join us at the AccessUSA Conference in Philadelphia
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The medical needs of individuals with rare
diseases go far beyond prescription reimbursement. Since 1987, NORD has been a leader in developing and implementing patient assistance programs designed specifically for rare patients.
Companies looking to maximize medication access and stay abreast of patient assistance industry standards are invited to come meet our RareCare™ Patient Services team at the Patient Assistance & Access Programs conference March 19-21 in Philadelphia, some of whom will be speaking at the event. Use our exclusive code 24NORD20 to save 20% when you book.
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Orphan product and rare disease therapy
leaders can also use our exclusive code to attend the adjoining Rare Disease Summit. Use code 24NORD20 to save 20%. on tickets.
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Register for the TCS New York City Marathon
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Runner applications are now open for the TCS New York City Marathon on November 4, 2024! NORD is excited to be an official charity partner for the TCS New York City Marathon for the 9th year in a row. Click here to apply.
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Join Us at the Denver Colfax Marathon
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We still have spots
available for people interested in running in the Denver Colfax Marathon! Be sure to register here soon and select the National Organization for Rare Disorders as your
charity of choice.
Rare Kids Run! NORD has been accepted to the Fly Feet program! We have 10 FREE spots for kids with rare diseases in the 5k. With limited spaces, be sure to apply for your child by April 1. These will be first come, first serve, and parents will be shared a code to register their child by April 10. Apply today!
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Sign up to join us for one of the cheer sections for the Boston Marathon, Denver Colfax Marathon, Marine Corps Marathon and TCS New York City Marathon. Fun swag and signs provided to
cheer on our runners!
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Rare Disease Legislative Updates From Congress
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NORD has been hard at work on Capitol Hill monitoring rare disease legislation and advocating for the needs of our community. Here's a rundown of everything you need to know right now and how you can help, starting by taking
action on our policy alerts here.
Last month, both the RARE Act (H.R.7383) and the Creating Hope Reauthorization Act of 2024 (H.R.7384) were introduced in the House of Representatives.
The Creating Hope Reauthorization Act of 2024 would prevent a lapse in authorization for the Rare Pediatric Disease Priority Review Voucher (PRV) program, a crucial incentive to develop therapies for this particularly challenging patient population. Reauthorizing this vital program before the September 30, 2024, deadline would maintain an important tool in ongoing efforts to address the significant unmet treatment needs of the pediatric rare disease population. NORD is thankful to Reps. McCaul, Eshoo, Bilirakis, Barragan, Burgess, and Trahan for introducing the bill and for their long leadership in this space.
The RARE Act cements the Food and Drug Administration’s longstanding interpretation that orphan drug exclusivity is awarded based on FDA approved indications, providing critical clarity to support robust rare disease drug development. NORD thanks Rep. Bilirakis and Rep. Matsui for their continued leadership in rare diseases and Senator Baldwin and Senator Braun for their continued leadership on companion legislation (S.1214) in the Senate.
Contact your Representative TODAY and urge them to cosponsor these bills that would help improve access to safe and effective treatment and necessary medical care for rare disease patients!
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Prescription Drug Affordability Board (PDAB) Legislation Passes the General Assembly of Virginia
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In February, both bodies of the General Assembly of the Commonwealth of Virginia passed legislation, (SB274 (Deeds)/HB570 (Delaney), which would create a Prescription Drug Affordability Board (PDAB) in the State. If signed into law, this Board would be required to set an “upper payment limit” (UPL) for the cost of prescription drugs they determine create affordability challenges to health care systems or lead to high out-of-pocket costs to patients within the Commonwealth. Ahead of its movement, NORD sent a letter to the bill sponsor and Members of relevant Committees containing recommendations to ensure the unique needs of the rare disease community and orphan drugs are appropriately taken into consideration in the context of the legislation.
Similar pieces of legislation have been proposed in several states across the country, and NORD continues to monitor bill activity and engage with legislators to ensure the voice of the rare community is heard at every state capital!
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Rare Disease Advisory Council (RDAC) Updates
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The bipartisan RDAC legislation (HB2758 (Hernandez, A) in Arizona passed its first hurdle on February 15, passing unopposed out of the House Committee on Health and Human Services. We look forward to
working with the author to continue the momentum!
Meanwhile, California’s RDAC bill was introduced on February 14. Stay tuned for updates as the bill, Assembly Bill 2613 (Zbur), moves through the legislature.
If you would like to get involved in RDAC efforts, email RDAC@rarediseases.org.
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Earn Your Certificate of Completion Now:
Drug Development Education Series, Part One
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Did you know drug development begins long before the first patient enrolls in a clinical trial? Learn about the drug development process through NORD’s free online course series, "Rare Disease Drug Development: What Patients and Advocates Need to Know," available in both English and Spanish! This course is free and open to all and can be completed at your own pace, even on your mobile device.
Part One: Foundations and Pre-Clinical Research focuses on the importance of the pre-clinical period and how patients can participate – start learning today and work toward your certificate of completion!
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New and
Updated Rare Disease Reports
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NORD's comprehensive Rare Disease Database® offers vital information on more than 1,300 rare diseases including their symptoms, causes, treatments, clinical trials, and sources of help such as patient advocacy organizations. NORD's Rare Disease Database is a leading source utilized by patients, physicians, researchers, and regulators to understand and treat rare disorders.
NORD's Educational Initiatives team added new reports on IQSEC2-Related Disorder and Multicentric Osteolysis Nodulosis and Arthropathy Spectrum and updated 16 reports in the Rare Disease Database®.
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The Global
Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry
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Anyone who has been diagnosed with paroxysmal nocturnal hemoglobinuria, in the US or internationally, is welcome to join the Registry and contribute to a better understanding of PNH. The surveys are
brief and can be done at your own pace. Make your mark on research with the Global PNH Patient Registry!
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B-Cell Malignancies
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Beigene is sponsoring a dose-escalation and expansion study of the bruton tyrosine kinase targeted protein degrader BGB-16673 in patients with B-cell malignancies including marginal zone lymphoma, follicular lymphoma, non-Hodgkin lymphoma, Waldenström's macroglobulinemia, chronic lymphocytic leukemia, small lymphocytic lymphoma, mantle cell lymphoma and diffuse large B-cell lymphoma. Learn more.
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Hepatocellular Carcinoma-Cholangiocarcinoma
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The National Cancer Institute is conducting a phase 2 study to compare the effects of adding bevacizumab and atezolizumab to gemcitabine and cisplatin (chemotherapy) versus chemotherapy and atezolizumab in treating patients with combined hepatocellular carcinoma-cholangiocarcinoma that cannot be removed by surgery or that may have spread from where it first started. Learn more.
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Waldenström's Macroglobulinemia
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Beigene is sponsoring a study to evaluate the safety and efficacy of the BCL2 inhibitor BGB-11417 in patients with relapsed/refractory Waldenström's macroglobulinemia. Learn more.
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See How RDCA-DAP
Can Support Your Work
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Drug development can be extremely
challenging for rare diseases because of small patient populations and a limited understanding of how these diseases can manifest and progress.
The Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®) brings together the experiences of patients and the clinical data of researchers to improve our shared understanding of rare diseases and accelerate clinical drug development.
Any questions about RDCA-DAP or input into the project can be sent to: rdcadap@c-path.org
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UpcomingProfessional Development Series Webinar
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Register now for the next webinar in the RDCA-DAP Professional Development Webinar Series: “Improving Data Collection
for Rare Epilepsies: Case example from the TSC Natural History Database” on Thursday, March 14. This webinar will discuss how better standardization of data and data collection is needed in rare epilepsies.
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View Webinar: Disease Progression Modeling
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Disease progression modeling synthesizes
statistics with disease knowledge and data to inform predictions and understanding of disease course in populations and subpopulations. This is commonly used in model-informed drug development.
Using examples from rare and orphan diseases, this webinar, "Understanding disease progression models: What are they, why are they useful, and how are they applied in rare and orphan disease? A high-level overview," breaks down the high-level ideas behind disease progression models.
View this webinar on demand now.
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New Data Contribution Agreement
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C-Path recently signed a Data Contribution Agreement with ML Bio Solutions, who will contribute Limb-Girdle Muscular Dystrophy data for the first time to the platform. More information to come.
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RDCA-DAP Team at Upcoming Conferences
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Members of the RDCA-DAP team will be at the Rare Neurodegenerative Disease Efforts Under the ACT for ALS hybrid meeting. If you're attending, come say hello!
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FEATURED NEWS FROM OUR MEMBERS
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Register for the
Wake Up Narcolepsy Summit
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Register now for the 2024 Wake Up Narcolepsy National Summit in Seattle! Join Wake Up Narcolepsy to hear from leading experts in the field and people living with Narcolepsy and make connections with those who understand. This free, one-day hybrid conference in Seattle, WA is for people with Narcolepsy, their families, educators, clinicians and researchers. Leading experts, such as Dr. Emmanuel Mignot, Dr. Michael Grandner & Dr. Anne Marie Morse, and community members will share about the latest research and treatment options, impact of Narcolepsy, managing mental health, advocating for yourself and how to live a full life with Narcolepsy.
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Register:
Ehlers-Danlos Society ECHO Summit
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The Ehlers-Danlos Society is delighted to announce its next educational event taking place on Saturday, March 16, 2024. The EDS ECHO Summit: Emergency Care is a virtual event that addresses the unique challenges and considerations in providing emergency care for individuals with Ehlers-Danlos syndromes (EDS) and hypermobility spectrum disorders (HSD). The event welcomes healthcare professionals, individuals with EDS or HSD, and their caregivers, for sessions covering various topics to enhance awareness, preparedness, and effective management during emergencies. This event has been
approved for up to 6.75 CME/CEU/CE credits for healthcare professionals to claim for sessions attended LIVE.
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Register:EDS ECHO Pediatrics North America
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Registration is now open for the next EDS ECHO Pediatrics North America program commencing April 2024. EDS ECHO Pediatrics North America is a virtual program for healthcare
professionals worldwide, across all disciplines who want to improve their ability to care for children and younger people with EDS, HSD, and associated symptoms and conditions. The program is composed of eight sessions, each consisting of expert presentations and case-based interactive sessions delivered virtually via the Zoom platform.
Each session is one hour in length and is led by Dr. Sarah Schwartz & Constance O’Connor from Toronto, Canada. Click here for more information about session topics and details.
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The
Ehlers-Danlos Society is Hiring!
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The Ehlers-Danlos Society is seeking a curator or person to take over the maintenance of the Ehlers-Danlos syndrome Sequence Variant Database. The database system is hosted by Leiden University Medical Center (LUMC) in the Netherlands using the Leiden Open Variation Database (LOVD) system. The task requires dedication, attention to detail, and a good understanding of database basics. In addition, a basic grasp of Hypertext Markup Language (HTML) would be advantageous.
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ASXL Rare
Research Endowment Grants
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The ASXL Rare Research Endowment is accepting travel grant applications for investigators who are early career and/or from diverse backgrounds to present work at the 2024 ASXL Research Symposium. Work must be related to the ASXL genes, mechanisms, or related disorders. Apply by Friday,
March 15.
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Upcoming Organic
Acidemia Association Forum
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The Organic Acidemia Association is excited to partner again with the FOD Family Support Group in hosting the upcoming FOD/OAA Family Forum. The event will be held on June 28-29, 2024 at the Wyndham Hotel University Center in Pittsburgh, PA.
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Upcoming NTM and
Bronchiectasis Conference
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NTM Info & Research is happy to announce its 2024 NTM and Bronchiectasis Patient Conference. After putting it on hold for 4 years due to the pandemic, they are pleased to provide an
educational and interactive program where patients can all connect in person again. The conference is scheduled for the afternoon of May 16, 2024, and the entire day of May 17, 2024, in San Diego, CA. The format is hybrid, with in-person sessions and virtual attendance for most sessions. Two of the planned sessions have interactive sections which will not support a live broadcast format. More details to follow soon.
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Upcoming
Hydrocephalus Association Conference
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Join the Hydrocephalus Association at the 18th National Conference in Tampa, Florida, on July 25-27! Don't miss your chance to hear from experts addressing medical,
educational, and social complexities of living with hydrocephalus. Register today!
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Improving the health and well-being of people with rare diseases by driving advances in care, research and policy. Learn more: rarediseases.org
Copyright ©2024 NORD – National Organization for Rare Disorders, Inc. All rights reserved.
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