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| Less Than One Month Until Rare Disease Day!
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Tuesday, February 28, 2023, is Rare Disease Day – a day to raise awareness of the 7,000+ rare diseases that affect over 300 million people globally. From wearing stripes to lighting up your office building as part of a global chain of lights, there are many ways to get involved and show your support!
Hosting an event? Let NORD help you spread the word by submitting it to our events calendar.
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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!
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| Get Involved With NORD's Advocacy Taskforce
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In 2023, we are continuing to make our rare voices and
priorities heard, and NORD is excited to present a new volunteer opportunity.
Are you passionate about rare diseases and public policy? Are you interested in getting more involved in federal or state policy, or both? Our National and Regional Taskforces will provide forums for volunteers to be more deeply involved in grassroots advocacy efforts and to fight for legislative solutions. The Taskforce is open to all, including experienced advocates and new volunteers, and we will offer you the training and tools you need to be successful.
If you’re interested in getting involved, sign up to receive
information about upcoming meetings and other advocacy opportunities.
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| ICYMI: The
Orphan Drug Act Turned 40
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January 4, 2023 marked the official 40th anniversary of the groundbreaking Orphan Drug Act (ODA) — legislation that incentivized drug companies to put more resources toward the research, development, and distribution of therapeutics for people with rare diseases.
The creation of the ODA and the founding of the National Organization for Rare Disorders (NORD) are forever intertwined. The rare patients and caregivers who
started the rare advocacy movement — which convinced lawmakers to introduce orphan drug legislation — knew their work was not done after the bill became law. They formed NORD to unify the voices of the unheard within the rare disease community under the premise that a rising tide could lift all boats. Read on to learn about the activists who started the rare disease advocacy movement and paved the way for the work we are doing today.
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| Apply For Our Rare Disease Centers of Excellence Program
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Applications are once again being accepted from medical institutions that wish to be considered as a designated site of our Rare Disease Centers of Excellence Program.
Potential candidates are leaders in the medical community, with strong ties and programs related to rare diseases. Applicants should be committed to working collaboratively and joining the Program with same spirit of enthusiasm and passion as the inaugural 31 sites.
Applications must be submitted by Thursday, February 23, 2023 at 11:59pm PT.
Do you or a loved one receive care at an exceptional medical institution? Share this information with your medical care team!
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| Join the 2023 NORD Running for Rare Team!
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NORD is an official charity partner of the 2023 TCS New York City Marathon! Interested in running? Applications are now being accepted for the NORD
Running for Rare team. Accepted runners will be notified on March 1, 2023.
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| Does Your State Make the Grade? Check Out Our 2023 Report Card
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Every year, NORD evaluates all 50 states and Washington, DC across nine key areas that are important to people with rare disorders. The annual NORD State
Report Card® serves as a tool for advocates and lawmakers to assess how their states are serving the rare disease community, and work toward meaningful policy change.
Curious how your state measures up? Visit our website to read the our newly released 2023 State Report Card now!
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| Looking to Grow Your Nonprofit? Check Out NORD's Learning Platform
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Did you know NORD has free, on-demand courses and educational resources to grow your nonprofit organization? Through our RareLaunch program, we share guides, templates and videos through an accessible, self-guided platform. These courses are a great way to learn the basics around nonprofit governance and rare disease research!
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| Watch NORD's New Animated Videos
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New animated videos in English and Spanish on Genetic Testing for Rare and Undiagnosed
Diseases have been added to NORD’s Rare Disease Video Library.
We are grateful to Horizon Therapeutics and Alnylam Pharmaceuticals for providing educational grants to make these videos possible. NORD is solely responsible for video content, produced in partnership with Osmosis from Elsevier.
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| Newly Updated: Rare Disease Reports
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| The Global Paroxysmal Nocturnal Hemoglobinuria Patient Registry
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PNH Patients Matter! The Global PNH Patient Registry is an interactive and dynamic resource that collects patient-driven data. Every patient’s story and experience are a unique, invaluable part of a disease’s natural history. Join now and let your data tell your story.
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| Amyotrophic Lateral Sclerosis (ALS)
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| Ionis Pharmaceuticals, Inc. is sponsoring a Phase 3 randomized study to evaluate the safety, efficacy, and pharmacodynamics of intrathecally administered ION363 in treating ALS patients with fused in sarcoma mutations.
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| Prothena Biosciences Ltd is sponsoring a Phase 3 multicenter, global, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of birtamimab in treating Mayo Stage IV patients with AL amyloidosis.
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| Amyotrophic Lateral Sclerosis (ALS)
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Ionis Pharmaceuticals, Inc. is sponsoring a phase 3, randomized study to evaluate the safety, efficacy, and pharmacodynamics of intrathecally administered ION363 in treating ALS patients with fused in sarcoma mutations. More.
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Ionis Pharmaceuticals, Inc. is sponsoring a phase 1-2a, open-label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intrathecally administered ION582 in treating patients with Angelman syndrome. More.
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| Primary Mitochondrial Myopathy
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| Reneo Pharma Ltd is sponsoring a Phase 2 randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of a 24-week treatment with REN001 in patients with primary mitochondrial myopathy.
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Rare Diseases Cures Accelerator -
Data & Analytics Platform News
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| Save the Dates For Our 2023 Webinars
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| Save the dates for the 2023 RDCA-DAP Webinar Series: April 20, May 18, June 22, July 20, October 19, and November 16. Topics will range from discussions on clinical outcome assessments and de-identification tools to informed consent considerations for patient registries and foundations, plus best practices for data collection in the rare disease space. Watch for more details to follow!
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| C-Path Welcomes New Associate Director of Data Science
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In January, the Critical Path Institute (C-Path) welcomed Nicole Vasilevsky, Ph.D., to the RDCA-DAP team as the Associate Director of Data Science for the Data Collaboration Center. In her first six months at C-Path, Nicole will work primarily on contributing to the data integration pipeline and leading the Ontologies, Standards and Metadata (OSM) team. Connect with Nicole here.
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| RDCA-DAP at Upcoming Conferences
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Members of the RDCA-DAP team will be at several conferences in February, including the 19th Annual WORLDSymposium 2023 for the Lysosomal Disease Annual Meeting.
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| See How RDCA-DAP Can Support Your Work
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| RDCA-DAP® is an FDA-funded initiative led by the Critical Path Institute that provides a centralized and standardized infrastructure to support and accelerate rare disease characterization,
with the goal of accelerating therapy development across rare diseases.
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| Welcome to Our New Members
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| We are excited to welcome the following patient organizations to the NORD membership networks! Learn more about our newest members.
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SHINE Syndrome Foundation was formed to represent and fight for the global DLG4 Synaptopathy community. The foundation organizes medical resources, works with researchers, has developed patient registries, raises funding for research, and drives awareness of SHINE Syndrome among the medical community
and the general public.
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Healing NET Foundation optimizes the care of those with neuroendocrine cancer through the education of and collaboration among physicians, health care providers, patients, and caregivers.
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wAIHA Warriors alleviates suffering and socioeconomic impact of wAIHA (warm Autoimmune Hemolytic Anemia) by fostering and facilitating collaboration in the areas of education, public awareness, research, and patient engagement.
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Shwachman-Diamond Syndrome (SDS) Alliance improves outcomes for all SDS patients, especially populations from diverse backgrounds who don’t have possible stem-cell donor matches. The alliance drives research and engages in advocacy and education.
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| Explore Sarcoidosis Volunteer Opportunities
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| The Foundation for Sarcoidosis Research is seeking dynamic, driven individuals impacted by sarcoidosis to apply to become Community Outreach Leaders and Support Group Leaders! These leaders will work together as part of volunteer teams serving areas close to FSR Global Sarcoidosis Clinics.
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| Registration for Family Camp is Now Open!
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Wake Up Narcolepsy (WUN) sponsors an annual camp, free of charge, for families of children and teens with narcolepsy. Join WUN in Scottsville, KY on September 22-24 for an unforgettable experience. Registration is now open! Learn more.
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| United MSD Foundation is Hiring!
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| The United MSD Foundation is seeking an Executive Director to lead and be responsible for ensuring the day-to-day management of the organization, and financial growth to fulfill the operational and research program goals and objectives.
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| Register for a CurePSP Community Conversations Webinar
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| CurePSP is hosting a Community Conversations webinar featuring Emma Wodwaski and Sanjay Geevarghese, who are both grandchildren affected by PSA and CBD. They will discuss how they draw inspiration from their relationships with their grandparents and offer their advice to people living with PSP, CBD, and MSA and their caregivers.
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| In-Person: Join the wAIHA Warriors Annual Patient Meeting
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| The first ever wAIHA Warriors Annual Patient Meeting will take place on March 24-26 in New Orleans. The conference is specifically for patients and caregivers and includes information from patients, physicians, and researchers regarding wAIHA clinical trials, current treatments, access to care, federal and state advocacy efforts, and more! Free registration and travel stipends are available for patients and caregivers.
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| Join a Pemphigus and Pemphigoid Virtual Support Group
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| Connect virtually with other patients and caregivers at the International Pemphigus and Pemphigoid Foundation (IPPF) Mid-Atlantic Area Virtual Support Group Meeting on February 18 at 1:00 pm EST. Living with pemphigus or pemphigoid is hard enough, but when you feel alone it seems even harder. You are not alone! This is a free and virtual meeting.
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| Join A Virtual Hour of Play for Children in the Rare Community
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The Barth Syndrome Foundation, Children's Cardiomyopathy Foundation, National Neutropenia Network and Sudden Arrhythmia Death Syndrome Foundation have partnered with Project Sunshine to offer a virtual hour of play for children between the ages of 5-10 years old who have been diagnosed with a rare disorder and their rare siblings. This is a great way for children with a rare disease and their siblings to connect with others who are a lot like them, regardless of their diagnosis. To be join the meeting, use the password "sunshine" and this link.
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| MENA Organization for Rare Diseases Annual Meeting
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| MENA Organization for Rare Diseases and UAE Genetic Diseases Association will hold their 2023 Annual Meeting in Dubai from March 3-5.
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You received this email because you signed up for the NORD Newsletter or other NORD email subscriptions.
National Organization for Rare Disorders, 1900 Crown Colony Drive, Suite 310, Quincy, MA 02169, United States
Copyright ©2023 NORD – National Organization for Rare Disorders, Inc. All rights reserved.
Our mission: We improve 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.
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