No images? Click here Wishing Our Rare Community a Safe, Happy 2021 We are honored to be a part of the rare disease community and to serve patients and families. For this new year, our hope is that you and yours remain healthy, happy and safe. From your NORD team–Happy New Year! Featured News and EventsINTERESTED IN BECOMING A SPEAKER? We are looking for enthusiastic and experienced individuals to share their insights at our 2021 Living Rare Forum! Submit your speaker interest application before the deadline–Friday, January 15. WHO INSPIRES YOU? In addition to seeking terrific contributors to the 2021 Living Rare Forum, we are also accepting in nominations for the people, groups and organizations who you've noticed going above and beyond for the rare disease community to be recognized at this year's Rare Impact Awards. Nominations are due Friday, January 15. HOW WILL YOU SHOW YOUR STRIPES? NORD's #ShowYourStripes campaign takes its cue from the majestic zebra, the unofficial symbol of rare diseases in the United States. Celebrate by showing your stripes and sharing how being a part of the rare disease community makes you feel proud, strong and unique! With the countdown officially started, visit our website to see how you can support Rare Disease Day 2021! EVENTS AND RESOURCES NOW AVAILABLE ON-DEMAND NORDpod: The Voice of Rare Disease Listen and subscribe to season one of NORDpod, the newly launched rare disease podcast from NORD! Episodes are available wherever you listen to podcasts. Please rate and subscribe. Continuing Medical Education Sessions NORD and PlatformQ Health are pleased to bring you accredited, accessible, digital courses on rare disease education. Available sessions are appropriate for patients and providers. RareLaunch NORD has supported and helped to build new disease-specific organizations since our inception. Through our RareLaunch program you can benefit from that experience and our capacity building resources. NORD Assistance Programs NORD provides programs to help rare disease patients and caregivers access the support they need. These programs provide medication, financial assistance, respite and more for those who qualify. Advocacy NewsNORD’s Policy team advocates at the state and federal levels for policies to improve the lives of patients with rare diseases. This is just a quick look into our work from the past month; please visit our policy statements page to view the complete list of NORD’s policy work. PATIENT GROUPS URGE US DISTRICT COURT TO PREVENT DISCRIMINATION AGAINST LGBTQ AND LIMITED ENGLISH PROFIENCY PATIENTS NORD, along with more than a dozen patient groups representing millions of Americans with serious and chronic health conditions, have filed an amicus brief urging the US District Court for the Southern District of New York to preserve protections for lesbian, gay, bisexual, transgender and queer (LGBTQ) patients receiving health care services and restore translation notices for those with limited English proficiency (LEP) in the case of New York v. US Dept. of Health and Human Services. Read the press release. NORD JOINS CONSENSUS STATEMENT ON HEALTH EQUITY NORD’s President and CEO, Peter L. Saltonstall, along with more than 55 health organizations CEOs, signed onto a consensus statement and call to action urging health equity in the US. The statement acknowledges the issue of structural bias in the health care system and lays out some actions signers can take to begin to address disparities. Read the full letter. NORD JOINS LETTERS TO GOVERNORS OF OKLAHOMA, MONTANA AND NEW MEXICO ON CRISIS STANDARDS OF CARE PLANS As the number of coronavirus cases surges across the country, NORD continues to advocate for states to develop and implement crisis standards of care plans that do not discriminate against those with pre-existing conditions in the event of scarce resources due to COVID-19. In support of this effort, NORD joined coalition letters to the Governor of Montana requesting his state develop crisis standards of care plans that do not discriminate against those with pre-existing conditions, and to the Governors of New Mexico and Oklahoma requesting modifications to their draft crisis standards of care plans to ensure those with rare diseases are not discriminated against. NORD ASKS CONGRESS TO FUND RESEARCH, DEVELOPMENT AND VITAL PUBLIC HEALTH AGENCIES On Thursday, December 3, NORD joined more than 70 organizations in calling on Congress to support science and innovation by adequately funding pivotal public health agencies such as the National Institutes of Health (NIH) for 2021 and providing funding to reboot critical research in the COVID-19 relief package. Read the full letter. Subsequently, the latest COVID-19 relief and 2021 Appropriations package includes a four-year reauthorization of the Rare Pediatric Disease Priority Review Voucher program, a surprise medical billing fix, a $43 million dollar increase in funds for the Food and Drug Administration, and a $1.25 billion dollar increase in funds for the National Institutes of Health. PROJECT RDAC UPDATES
NORD SUBMITS COMMENTS TO CDC'S ADVISORY COMMITTEE ON IMMUNIZATION PRACTICES On Friday, December 18, NORD submitted comments to the Center for Disease Control and Prevention (CDC) Advisory Committee on Immunization Practices (ACIP) in advance of their meeting on Sunday, December 20 on vaccine prioritization. Currently, there is not sufficient data to know the extent to which those with a rare disease are at risk for severe complications from contracting COVID-19. Therefore, NORD urged the CDC to ensure that rare disease patients are given consideration when determining vaccine prioritization and allowing a rare disease patient’s health care provider to make a determination of a patient's risk and recommend vaccination at the appropriate time. Patient ResourcesFIVE UPDATED RARE DISEASE REPORTS Over the last month, NORD's Educational Initiatives team updated five rare disease reports in the Rare Disease Database®. Patient RecruitmentNORD NATURAL HISTORY OF METACHROMATIC LEUKODYSTROPHY (HOME) STUDY Hosted by NORD’s IAMRARE™ platform, the HOME Study enables patients and caregivers to contribute directly to research from the comfort and safety of their home, without the demands and challenges of traveling to a study site. The study will provide dynamic data collection and a new framework for building regulatory-grade rare disease natural history studies. UNDIAGNOSED RARE DISEASE REGISTRY With the Undiagnosed Rare Disease Registry, NORD’s goal is to provide a bird’s eye view of the undiagnosed landscape so we can better support patients and aid in the development of improved diagnostic methods and treatments. By participating in the registry, you are helping to drive research forward. Make a difference by signing up for the Undiagnosed Rare Disease Registry. KRABBE COMMUNITY UNITED RESEARCH AND ENGAGEMENT STUDY The Krabbe Community United Research and Engagement Study (KrabbeCURES), launched the largest-ever study to research Krabbe disease that damages the white matter (myelin sheath), as well as the rest of the central nervous system (CNS) and the peripheral nervous system, which consists of the nerves outside the CNS. Krabbe disease currently has no cure. The new study, KrabbeCURES, creates a platform for patients around the world to share information about Krabbe disease. Its purpose is to build an international resource to be used by scientists in future research. Immune thrombocytopenia (ITP) Principia Biopharma, Inc. is sponsoring a study of rilzabrutinib in patients with persistent or chronic ITP, with an average platelet count of <30,000/μL on two counts at least five days apart in the 14 days before treatment begins. More. Mantle cell lymphoma PrECOG, LLC is sponsoring a study to evaluate the effectiveness and potential side effects of ixazomib combined with ibrutinib chemotherapy in treating patients who have relapsed or refractory mantle cell lymphoma. More. PrECOG, LLC is sponsoring a study to evaluate the effectiveness and potential side effects of venetoclax combined with bendamustine and rituximab chemotherapy in treating patients ages 60+ who have mantle cell lymphoma. More. Thymidine kinase 2 (TK2) deficiency Modis Therapeutics, Inc. and Zogenix, Inc. are sponsoring a study to evaluate the efficacy and safety of MT1621 in patients with genetic confirmation of TK2d who have never received MT1621 or nucleos(t)ides before entering the study. More. RDCA-DAP News
Member News NORD is pleased to welcome one new patient advocacy group to the growing family of NORD member organizations. The Bonnell Foundations' mission is to give emotional and/or financial support to parents who have a loved one with cystic fibrosis. Their hope is that one day no cystic fibrosis parent will experience the pain of losing a child to this disease. CACNA1A Foundation The CACNA1A Foundation has announced a seed grant of $50,000 for a research project that shows promise for contributing to the scientific advancement and understanding of CACNA1A variants. Deadline for applications: Monday, February 1. More. CMTC-OVM CMTC-OVM invites you to tune into their upcoming webinar on vascular malformations and overgrowth with Dr. Christianne van Nieuwenhoven on Saturday, January 16. More. CurePSP CurePSP will be hosting a virtual International Research Symposium February 4-5 to bring together international investigators to discuss the latest scientific advances relevant to neurodegeneration with a particular focus on rare tauopathies such as PSP and CBD. The Symposium welcomes all researchers with an interest in preclinical and clinical neuroscience. More. Illness Challenge Foundation The Illness Challenge Foundation is proud to share the China Orphan Drugs Emergency Aid Project During COVID-19 Action Report. This report summarizes the task force ICF formed with China Alliance of Rare Diseases (CHARD) to respond to the strongest needs from the rare disease community. More. National Health Council Have you been hearing about Real-World Evidence (RWE) and Real World Data (RWD) and wondering what they are or how they might impact patients and care? The National Health Council and the Duke-Margolis Center for Health Policy are excited to announce their training series introducing RWD and RWE through March 2021! More. Siegel Rare Neuroimmune Association SRNA invites you to check out their Ask the Expert podcast on "MOG Antibody Disease: Adult and Pediatric Presentations" in collaboration with The MOG Project. The experts define MOG antibody disease, discuss the long-term effects and management of the disease and provide the acute and preventative treatments for MOGAD. More. Take a listen to SRNA’s ABCs of NMOSD podcast on “Psychosocial Aspects of NMOSD: Anxiety, Depression, and How to Cope” with Katherine Chapman, LCSW, CRND. She explains the ways in which NMOSD can affect mental health and describes different coping mechanisms and strategies for improving mental health, including therapy. More. SRNA shares an Ask the Expert podcast on “How to Manage Visual Issues” with Amanda Aaron, OTD and Deb Nikkila, MOT, OTR/L. The experts describe the purpose of low vision rehabilitation, the resources available for people experiencing visual issues and respond to questions regarding symptoms. More. Take a listen to the discussion about COVID-19 vaccinations and the implications for those with rare neuroimmune disorders between Dr. Benjamin M. Greenberg, a member of SRNA’s Board of Directors and Medical and Scientific Council, and GG deFiebre, SRNA’s Associate Director of Research and Education. More. The Bonnell Foundation The Bonnell Foundation is looking for a volunteer to do approximately five hours of work on their social media platforms. If interested, email thebonnellfoundation@gmail.com detailing why you would like to help and your experience. There is a possibility for this to become a part-time position in 2021. More. The Desmoid Tumor Research Foundation The Desmoid Tumor Research Foundation (DTRF) is looking to hire a US-based Research Director for the DTRF Coordinating Center. This Center will integrate and coordinate research and data across the DTRF investigators, expand the diversity of the research community and facilitate the exchange of scientific information. More. Uplifting Athletes Uplifting Athletes shares the Young Investigator Draft, which is the result of their ongoing commitment to cultivating resources that accelerate scientific advancements for rare disease treatments and potential cures while facilitating the next generation of rare disease researchers. The request for proposal deadline is Monday, January 11. More. |