Rare Disease – April 2023 eNews

Join us as we reimagine the future of rare in celebration of NORD's 40th anniversary and the 2023 Rare Impact Awards. This event will take place on Thursday, May 4, 2023, at 6:00pm ET at the Smithsonian National Portrait Gallery in Washington, DC. This special evening will recognize NORD's legacy and commitment to rare diseases, and honor those individuals, organizations, and companies doing remarkable work on behalf of the rare disease community.

Just announced! Emmy Award-Winning NBC News Journalist Peter Alexander will be hosting the celebration. Learn more about our incredible host at rareimpact.org.

View our distinguished 2023 Rare Impact Award honorees, including our founder and past President, Abbey S. Meyers on our website here.

Join NORD for the Living Rare, Living Stronger Patient and Family Forum on Saturday, May 6, 2023, hosted at the Renaissance Downtown Hotel in Washington, DC.

We will bring the rare disease community together in our nation’s capital for a day focused on learning, networking, and fun! Attendees will hear real stories from our community and gain practical knowledge to help them live their best life while navigating their rare disease journey.

This year’s program will include sessions such as Living with Uncertainty and Rare Breakthroughs: Hope Now and on the Horizon. Check out the online agenda here to learn more.

The 40th anniversaries of the Orphan Drug Act and NORD remind us that every person in the rare disease community has the power to shape the future and make a lasting impact.

As we picture the next ten years, we want to hear what you (re)imagine is possible for you and the entire rare community. What would make a difference in your rare journey, or a loved one's? What changes could empower you to live your fullest and best lives?

Share your vision and ideas today, and post on social media with the hashtag #ReimagineRare. Your voice is your gift to our community!

NORD is excited to be hosting a series of in-person and virtual community listening sessions exclusively for Latino/a/x people affected by rare diseases in the United States. We invite you to learn more and spread the word about this important opportunity happening throughout April, May, and early June.

These sessions are private events and personal information will remain confidential. They are also family-friendly, so individuals related to registered attendees are welcome to attend. Please note there are limited spots available for the in-person listening sessions and registration is required.

The in-person sessions will take place in New York City, Houston, and the DC Metro Area, with virtual options in each of those locations as well. There is also a national virtual listening session that any Latino/a/x rare patients in the US are welcome to attend.

If you, yourself, are interested in attending, you can submit your interest in a listening session by registering here.

For the first time, as part of the Inflation Reduction Act that passed last year, the Centers for Medicare & Medicaid Services (CMS) will be able to negotiate the prices of certain prescription drugs. The Medicare Drug Price Negotiation Program could have significant impacts on the more than 25 million people living with a rare disease and the ability for some to afford needed treatments, but could also impact broader rare disease drug development.

NORD is taking a leading role to ensure the law ultimately benefits patients and families living with rare disease, and we need your help. The government is seeking public comments between now and Friday, April 14. Learn more about NORD's position and submit your feedback here.

Join us for the April meetings of NORD’s Regional Policy & Advocacy Taskforces! These meetings will provide a platform for individuals to work with other rare disease advocates in their region to advance policies that benefit the rare disease community. The April Regional Taskforce meetings will take place at the time and dates listed below:

Region A: Monday, April 3 at 10:00 AM MST (9:00 AM PST)

Region B: Wednesday, April 5 at 3:00 PM CST (2:00 PM MST)

Region C: Thursday, April 6 at 12:00 PM EST (11:00 AM CST)

Region D: Tuesday, April 4 at 12:00 PM EST

No prior advocacy experience is necessary to join Taskforce meetings! We will provide you with the training and tools you need to be a successful rare disease advocate. You can learn more about the Taskforce, how to participate and which region you live in at this webpage and register for a Regional meeting here.

Be sure to save the date our next National Taskforce meeting on Tuesday, May 23 at 4:00pm ET. More details on how to register will be available soon.

NORD’s annual State Report Card is a tool for state advocates and lawmakers to assess how all 50 states are serving the rare disease community, and to aid in advocating for meaningful policy change. This year’s State Report Card was compiled using data through November 2022 and provides a detailed analysis of each state’s performance on nine major policy areas of importance such as newborn screening, Medicaid eligibility, RDAC formation, and prescription drug out-of-pocket cost protections.

Curious how your state measures up? Visit our website to read the full report card now!

NORD is pleased to announce that four patient advocacy organizations will be taking the major step forward of joining the IAMRARE community and submitting data to RDCA-DAP starting next month:

CureARS, which promotes research for the 19 mitochondrial ARS (mt-aaRS) genes and unites advocacy efforts from across the individual disorders linked to those genes.

FamiliesSCN2A, which supports research and advocacy to improve the lives of those affected by SCN2A-related disorders around the world.

National CMV Foundation, which educates families and healthcare providers about congenital cytomegalovirus (cCMV), affecting 1 in 200 newborns.

Recurrent Respiratory Papillomatosis Foundation (RRPF), which aims to make information available and hope possible for patients and families living with recurrent respiratory papillomatosis (RRP).

These four organizations were selected based on their organizational capacity and readiness to begin quickly submitting their existing data to the RDCA-DAP and taking advantage of this opportunity.

Read our full announcement to learn about the history of each of these organizations and the exciting ways they will deepen the RDCA-DAP's potential.

The CureDuchenne FUTURES National Conference is an annual event focused on bringing education, connection, and hope to individuals and families affected by Duchenne muscular dystrophy. This year’s event will be held April 20-23 in San Diego, CA, and the theme is BRIGHT FUTURES, placing importance on the acceleration of critical research, improvement of care and quality of life for all, and fostering a community that supports one another and takes on the future together. FUTURES attendees are guaranteed exclusive access to important updates on therapeutic research, thoughtful discussions on the emerging approaches to care that enhance quality of life, a wealth of valuable resources, and time well-spent with the Duchenne community. This conference is family-friendly, so bring the whole team! Learn more.

The 2023 CMT Patient & Research Summit on Saturday, November 4 will offer life-improving presentations on living well with CMT and will provide a comprehensive update on current STAR research. Spend the day learning how to manage CMT and hear how you can be involved in advancing CMT research through clinical trials and studies. Learn more.

With the goal of expanding our commitment to improving care for all people living with Charcot-Marie-Tooth disease, the CMTA will be hosting a 2023 Clinical Summit for health professionals on Friday, April 21 from 10-2 EST. Eight in-depth accredited sessions will be offered for medical teams in the following areas: neurology, genetic testing and counseling, pulmonology, orthopedic surgery, physical therapists, occupational therapists, clinical studies/patient recruitment, social workers. CMTA is partnering with the best disease experts in the industry to create an incredible day of education. Learn more.

TGCT Support and SPAGN have collaborated to bring awareness to the new and only consensus guideline for Tenosynovial giant cell tumor (TGCT), a rare tumor disease impacting a joint, bursa, or tendon sheath. TGCT is a debilitating, life-limiting neoplastic disease that has distinct subtypes impacting prognosis, treatment, and quality of life. The diffuse form of TGCT is highly recurrent and difficult to treat. Prior to this guideline, no standard was available and globally, patients were treated very differently. This consensus from 50 experts and 2 patient advocacy organizations provides the basis for harmonization in diagnosis, treatment, monitoring, and more based on level of evidence and provider experience. Learn more.

NHF is thrilled to announce that the 75th Annual Bleeding Disorders Conference (BDC) will take place from August 17 to 19, 2023, in National Harbor, Maryland, at the beautiful Gaylord National Resort & Convention Center! Join NHF for three full days of educational sessions, networking opportunities, and access to our exhibit hall. Your registration includes entrance to the Opening Session, Awards Luncheon, and the exciting Final Night Event. We also have an Educational Kids Program available for children 12 years old and younger. Learn more.

The Castleman Disease Collaborative Network announced the launch of the ROADMAP drug repurposing tool! The ROADMAP tool is the culmination of the ROADMAP project pursued by the CDCN over the past two years, in partnership with the Chan Zuckerberg Initiative and Dr. Fajgenbaum’s new nonprofit, Every Cure. It is a 5-step easy-to-follow guide for how to repurpose drugs for rare diseases, with potential roadblocks highlighted at every stage and data from real world experiences. The interactive ROADMAP tool and the data insights are now available to explore! Learn more.

This year, Uplifting Athletes is empowering a new roster of NFL prospects with the opportunity to use either their NFL Combine or Pro Day workouts as a platform to give back to the rare disease community. Reps for Rare Diseases strives to raise funds and awareness for Uplifting Athletes’ mission and vision. Since its inception in 2015, more than 260 players have participated in Reps for Rare Diseases and have been able to raise over $350,000. When you partner physical exertion with a cause-driven mission you get high-powered, compassionate athletes who are willing to go above & beyond for the rare community. Learn more.

April is MOGAD Awareness Month. To shine a light on MOG antibody disease (MOGAD) and the journeys of all those diagnosed with this rare disorder and their loved ones, the Siegel Rare Neuroimmune Association (SRNA) will be sharing stories, resources, facts, and helpful tips during the entire month of April. SRNA will be kicking off MOGAD Awareness Month with MOGAD Together. Join them online for a two-day international event on April 6 & 7. Learn more about MOG Antibody Disease, connect with others in the rare neuroimmune disorders community, and raise awareness for MOGAD. Learn more.

The Foundation for Sarcoidosis Research (FSR) is proud to announce their Inaugural Sarcoidosis Crystal Awards Gala: Celebrating Connections, Collaboration, and Catalyzing Research will take place on May 24, 2023, in Washington, DC. The Gala will bring together the sarcoidosis community for an evening celebrating clinicians, researchers, and advocates from around the globe who are leading the charge to advance sarcoidosis research and carve the path towards better treatments and a cure. Early Bird ticket pricing is available until April 15. Learn more.

The United MSD Foundation seeks an enthusiastic, experienced, and mission-driven Marketing and Communications Specialist to join their team as they advance ever closer to a cure for MSD. The ideal candidate will take initiative, be resourceful, and exhibit a problem-solving mindset. The successful candidate must be able to communicate effectively with a diverse range of stakeholders from patient families to funders to the scientific community. Submit your resume with a link to your portfolio of work or 3 samples of marketing and communications work to Sarah Cortell Vandersypen, Executive Director, at svandersypen@curemsd.org. Apply by April 6 for priority consideration. Learn more.

The Platelet Disorder Support Association (PDSA)'s College Scholarship Program offers financial assistance to senior high school students, college students, or adults interested in continuing education who are suffering from immune thrombocytopenia (ITP) or a similar platelet disorder. Awards range from $250 to $1,500, and the top scholarship winner also receives two complimentary registrations to attend the ITP Conference 2023 in Chicago, IL from July 21-23. Applications will be accepted through April 14, 2023. Learn more.

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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 at: rarediseases.org

NORD is a registered 501(c)(3) charity organization. Please note that NORD provides this information for the benefit of the rare disease community. NORD is not a medical provider or health care facility and thus can neither diagnose any disease or disorder nor endorse or recommend any specific medical treatments. Patients must rely on the personal and individualized medical advice of their qualified health care professionals before seeking any information related to their particular diagnosis, cure or treatment of a condition or disorder.