No images? Click here Featured News and Events THE NORD SUMMIT IS ONLY WEEKS AWAY! Registration is open for the NORD Rare Diseases and Orphan Products Breakthrough Summit! Join us virtually on October 18 and 19 to unlock new insights alongside experts from across the globe, network in an engaging and supportive environment, and discuss critical topics in the rare disease field. ATTEND EXPERT-LED PANELS AND PLENARIES EVENTS AND RESOURCES NORD Assistance Programs NORD offers 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. 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 both patients and providers. NORDpod: The Voice of Rare Disease Listen to the first two episodes of NORDpod season three! Episodes are available wherever you listen to podcasts. Don't forget to rate and subscribe to us as well! Advocacy and Policy NewsTAKE ACTION Ask Congress to Support Rare Disease Patients in the Reconciliation Package! Congress is developing a $3.5 trillion legislative package, known as the Build Back Better Act, which could include several health care priorities important to the rare disease community. Reach out to your legislators today to share how this legislation should benefit the rare disease community. Read more and take action here! Tell Congress to Protect the Orphan Drug Tax Credit Today! The House Ways and Means Committee recently advanced their portion of the Build Back Better Act, which included language to amend the Orphan Drug Tax Credit in ways that would severely undermine efforts to ensure more patients have access to a safe and effective drug for their rare condition. NORD sent a letter, co-signed by 86 other rare disease organizations, to Senate and House reconciliation leaders highlighting robust opposition to the changes to the Orphan Drug Tax Credit and urging them to leave such provisions out of the final bill. Please take action and reach out to your legislators and ask them to leave the proposed changes to the Orphan Drug Tax Credit out of the final version of the Build Back Better Act! POLICY VICTORIES Virginia’s Rare Disease Advisory Council (RDAC) Appointments All members of the Virginia RDAC have officially been appointed. Congratulations to NORD’s Virginia volunteer State Ambassador, Jana Monaco, who has been selected to serve as the RDAC’s Vice-Chair. View the list of those appointed to the council here. California Victories Senate Bill 247, which will establish an RDAC, and Assembly Bill 347, which will implement common-sense patient utilization management protections, are both waiting Governor Newsom’s signature to become law! KEY POLICY UPDATES NORD is Proud to Partner with Other Patient Advocacy Organizations to Advocate for a National Paid Medical Leave Program Do you have a story to share about your experience with paid leave? Please email us at policy@rarediseases.org. Participate in an RDAC Coalition Meeting Do you want to get more involved in rare disease advocacy in your state? Join us for the next Project RDAC meeting in your state to provide valuable input into the ongoing effort! Learn more here. NORD sends joint letter to the Senate Labor Health and Human Services, Education, and Related Agencies Appropriations Subcommittee Requesting Support for Rare Disease Programs in FY22 On Wednesday, September 15, NORD, along with the Muscular Dystrophy Association (MDA), and Friedreich's Ataxia Research Alliance (FARA) sent a letter to the Senate Labor, Health and Human Services, Education, and Related Agencies Appropriations Subcommittee requesting increased appropriations for rare disease programs at the National Center for Advancing Translational (NCATS) Science in Fiscal Year 2022. Patient & Caregiver ResourcesSIX RARE DISEASE REPORTS UPDATED Over the last month, NORD's Educational Initiatives team updated six rare disease reports in the Rare Disease Database® and added four new reports on Congenital Tracheomalacia, Tracheobronchomalacia, Pendred syndrome and Perrault syndrome. Patient RecruitmentNORD NATURAL HISTORY OF METACHROMATIC LEUKODYSTROPHY (HOME) STUDY The National Organization for Rare Disorders is proud to announce the NORD Natural History of Metachromatic Leukodystrophy (HOME) Study, a brand-new initiative with support from FDA CBER! The HOME Study, hosted by NORD’s IAMRARE™ Program, enables patients and caregivers to virtually 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. THE GLOBAL PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) PATIENT REGISTRY PNH Awareness week is coming up on October 10th-17th! Raise awareness and enhance research efforts by joining the Global PNH Patient Registry! THE TATTON BROWN RAHMAN SYNDROME COMMUNITY REGISTRY NORD is pleased to announce the launch of the Tatton Brown Rahman Syndrome Community Registry, a collaborative effort between Tatton Brown Rahman Syndrome Community and NORD to study Tatton Brown Rahman Syndrome (TBRS). Tatton Brown Rahman Syndrome Community supports research on rare diseases and how they progress over time - 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. ACUTE MYELOID LEUKEMIA (AML) Lynk Pharmaceuticals is sponsoring a multicenter, open-label, phase 1 study designed to evaluate safety and tolerability of multi-kinase inhibitor LNK01002 in patients with AML. More. MYELOFIBROSIS Lynk Pharmaceuticals is sponsoring a multicenter, open-label, phase 1 study designed to evaluate safety and tolerability of multi-kinase inhibitor LNK01002 in patients with primary myelofibrosis, or myelofibrosis due to polycythemia vera. More. RDCA-DAP News
Member News Chondrosarcoma Foundation's mission is to educate the public about chondrosarcoma, to support and advocate for patients and families, and improve treatment and care. The International FOXP1 Foundation's mission is to build a global community that empowers and supports families and individuals with FOXP1 syndrome by sharing knowledge, inspiring hope, encouraging research, and raising awareness. Superior Mesenteric Artery Syndrome Research Awareness and Support (SMASRAS) is dedicated to promoting health care opportunities for under-insured individuals and educating both the medical community and the public about SMAS to encourage early diagnosis. FACES - The National Craniofacial Association serves children and adults throughout the US with severe craniofacial differences resulting from birth defects, injuries, or disease through support in client travel, and public awareness, and understanding. Narcolepsy Network's mission is to educate and inform individuals with narcolepsy about the condition, available treatments, and symptom management so they may be empowered to achieve the highest quality of life possible. Wings of Hope changes and saves lives through the power of aviation, working on a global scale to help vulnerable communities access the basic resources essential for human dignity. AMERICAN PORPHYRIA FOUNDATION The American Porphyria Foundation is hosting “Heme Biosynthesis and the Porphyrias 2021: Consensus Diagnoses, Variant Disorders, New & Emerging Therapies,” to be held October 15-17 in Chicago. More. APBD RESEARCH FOUNDATION The APBD Research Foundation is seeking a remote, part-time Communications and Development Manager (CDM) to help join their team during this crucial period of growth in the community. More. CHARCOT-MARIE-TOOTH ASSOCIATION The Charcot-Marie Tooth-Association is hiring a Chief Scientific Officer to lead their research program. More. The Charcot-Marie-Tooth Association is hiring a Digital Marketing Manager. To learn more: More. CMTC-OVM CMTC-OVM’s global members conference for patients with vascular malformations and their families is Saturday, October 30 and will be both on location as well as online. Speakers from different countries will be present and patients can get personal medical advice. More. CUREDUCHENNE The CureDuchenne 2021 FUTURES National Conference will take place October 8-10. It is a two-day event focused on bringing education, resources, and connection to the Duchenne community in a family-friendly environment. More. CUREPSP CurePSP, Inc. will be hosting an in-person and online film debut and cocktail reception for "Train Keeps a Rolling" on October 6 at City Winery in New York. It honors the life of the world-renowned guitarist Jeff Golub and his wife, Audrey Stafford. Jeff suddenly lost his sight at the age of 56 because of a rare neurological disorder, progressive supranuclear palsy (PSP). More. HCU NETWORK AMERICA HCU Network America and HCU Network Australia are announcing they are now accepting Expressions of Interest for their research program. More. IMMUNE DEFICIENCY FOUNDATION (IDF) IDF is seeking a Director of Science and Policy Communications who will be responsible for protecting the integrity and enhancing the accessibility of information shared by IDF while ensuring IDF stays on the leading edge of innovative treatments and medical advancements. More. INTERNATIONAL FOUNDATION FOR GASTROINTESTINAL DISORDERS (IFFGD) The International Foundation for Gastrointestinal Disorders will be commemorating their 30th anniversary with a special virtual edition of the Norton Education Series (NES). The Nancy and Bill Norton Patient Education Series Event: IFFGD’s Thirtieth Anniversary Edition will be a mix of pre-recorded videos and a live feed and will take place November 6-7. More. INTERNATIONAL WALDENSTROM’S MACROGLOBULINEMIA FOUNDATION This year’s Virtual Educational Forum is a unique opportunity to learn from medical experts and connect with WM community members around the world. October will be Ed Forum Month, featuring informative sessions throughout the month. More. MOEBIUS SYNDROME FOUNDATION The Moebius Syndrome Foundation is looking to contract with an experienced conference and meetings planner to assist on a part-time basis for projects through July 2022. More. NARCOLEPSY NETWORK Join Narcolepsy Network's Virtual Annual Conference October 2-3. More. NATIONAL ADRENAL DISEASES FOUNDATION (NADF) NADF was excited to support the work of Dr. Patrick Reeves at the Walter Reed National Military Medical Center. Dr. Reeves' desire was to improve pediatric adrenal insufficiency literacy using pictograms. He enlisted NADF to help the effort. They created the Pediatric Adrenal Insufficiency Action Plan with Pictograms: a tool to help patients and families recognize adrenal insufficiency symptoms and take appropriate actions. More. NATIONAL ATAXIA FOUNDATION (NAF) The National Ataxia Foundation is seeking Letters of Intent for a NAF research grant. Grant types and funding vary by award type. NAF is committed to funding the best science relevant to hereditary and sporadic types of ataxia in both basic and translational research. More. NATIONAL TAY-SACHS & ALLIED DISEASES ASSOCIATION (NTSAD) NTSAD's annual Imagine & Believe reception will be held on Thursday, October 28 at the Royal Sonesta Boston. For those living outside the Boston area, there will be a free virtual Imagine & Believe event on Wednesday, November 3 from 7-8 pm ET. More. NTSAD is hiring their first Director of Clinical and Research Initiatives to engage with key stakeholders and focus on the discovery and development of treatments, diagnostics and technologies for Tay-Sachs, Canavan, GM1 gangliosidosis, and Sandhoff diseases. More. NTSAD is searching for its next Executive Director. They are seeking a passionate, experienced, and inspiring leader in this unprecedented time of progress and hope. More. SICKLE CELL DISEASE ASSOCIATION OF AMERICA (SCDAA) SCDAA named 14-year-old Ayana Lee Johnson the 2021-2023 SCDAA National Teen Ambassador. She was selected through a nationwide competition formerly known as the Poster Child Contest that started in 1976. Her reign will begin with an official coronation during SCDAA’s Annual National Convention October 12-16. More. SPINAL CSF LEAK FOUNDATION Registration is now open for Spinal CSF Leak Foundation's Fourth Annual Cedars-Sinai Intracranial Hypotension Symposium, which will have both virtual and live events in Los Angeles, CA, on Saturday, October 2. More. THE MAST CELL DISEASE SOCIETY Registration is open for The Mast Cell Disease Society's 24th National Conference. The conference will be held virtually October 13- 20 and will consist of lay-person-focused medical presentations, panel discussions, workshops, and support group activities. More. SIEGEL RARE NEUROIMMUNE ASSOCIATION (SRNA) In this Part VI of our Q&A series, Dr. Benjamin M. Greenberg, member of SRNA’s Board of Directors and SRNA’s Medical and Scientific Council, discussed COVID-19 vaccinations and the implications for those with rare neuroimmune disorders. More. Rebecca Whitney of SRNA was joined by Dr. Jayne Ness and Dr. Silvia Tenembaum for an ABCs of NMOSD podcast entitled, “Pediatric NMOSD – History and Diagnosis, Part I.” More. Dr. Hesham Abboud joined Dr. Chelsey Judge of The Connor B. Judge Foundation for an ABCs of NMOSD podcast entitled, “Navigating Treatment Options.” More. SLC6A1 CONNECT Rare disease patients are often lost in the diagnostic odyssey. Learn from two rare moms and a genetic counselor about whole genome sequencing and advocating for a loved one. More. |