Preview
Explore new grants, new research, highlights from the NORD Summit, and more.
 ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌ ‌
 
NORD banner with tagline "Alone we are rare. Together we are strong."
 
ABOUT |  NEWS |  EVENTS |  CONTACT |  DONATE
 
Graphic with the photo of a zebra's side and the text "Monthly Newsletter"
 

Featured News and Events

THANK YOU FOR A REWARDING NORD SUMMIT!
Thanks to all who joined us for the 2022 Rare Diseases & Orphan Products Breakthrough Summit! NORD welcomed more than 800 enthusiastic attendees to Washington, DC for two days of discussions and networking on challenges and opportunities in rare disease. Mark your calendar for next year's Summit, taking place Sunday, October 15 – Tuesday, October 17, 2023. Unable to join us this year, or want to revisit a session? On-demand access is now available through the event app. View more photos from the event.
 
APPLY NOW FOR NEW GRANT OPPORTUNITIES

NORD is pleased to announce $70,000 in grant funding for rare disease research! Grants will be awarded to qualified researchers to initiate small research studies or clinical trials which could be used to obtain funding from NIH, FDA, or other agencies; or to attract a corporate sponsor.


Three grants are available for research on Levy-Yeboa syndrome (LYS), megacystis microcolon intestinal hypoperistalsis syndrome (MMIHS), and autoimmune polyglandular syndrome type 1 (APS-1).
 

Policy and Advocacy News

FDA announces more than $38 million in rare disease research funding, but without swift congressional action, the grants program will lose authorization this December.
In his opening remarks for NORD’s Breakthrough Summit on Monday, October 17, FDA Commissioner Dr. Robert Califf announced that the Food and Drug Administration (FDA) was awarding more than $38 million in research funding to advance the development of medical products to treat rare diseases. The funding, which is part of FDA’s Orphan Products Grants Program, will support clinical trials, natural history studies, and regulatory science tools related to rare diseases. The Orphan Products Grants Program has supported clinical rare disease research since 1983 and facilitated the approval of more than 80 products. However, the program is currently only authorized by Congress to operate until Friday, December 16.

NORD is educating decision-makers and stakeholders about the importance of the program for the rare disease community and urging its long-term reauthorization as part of an end-of-year legislative package that is in development. Stay tuned for opportunities to share your support for this critical program!

 

Educational Resources from NORD

EXPLORE UPDATED RARE DISEASE REPORTS

NORD's Educational Initiatives team has updated the Rare Disease Database® with new reports on Idiopathic Pulmonary Hemosiderosis and Loeys-Dietz Syndrome. Visit the database now to learn more.

STUDENTS FOR RARE CONVENE AT SUMMIT
On Sunday, October 16, 19 Students for Rare leaders from across the country convened for an adjunct meeting before the NORD Summit. The students, who ranged from high school to graduate level, heard from guest speakers Vinaya Murphy MPH, MS, LCGC and Dr. Reena Kartha, MS, PhD. about the importance of rare diseases in undergrad and medical school curriculum. The students also presented innovative ideas for upcoming Rare Disease Day events, and collaborated with leaders from other institutions on how to expand advocacy reach. Participants gained invaluable insights to bring back to their chapters.
Students share ideas for awareness-raising activities on their campuses for Rare Disease Day 2023.

FDA News

FDA ANNOUNCES NEW GRANT AWARDS

The FDA has awarded 19 new grants and two new contracts totaling more than $38 million in funding over the next four years to support clinical trials, natural history studies and regulatory science tools related to rare disease, including ALS. Learn more.

TWO NEW DRUGS APPROVED BY FDA

Relyvrio (sodium phenylbutyrate/taurursodiol) has been approved to treat patients with amyotrophic lateral sclerosis (ALS). Relyvrio is manufactured by Amylyx Pharmaceuticals Inc. Read the press release.

Dupixent (dupilumab) has been approved to treat adults with prurigo nodularis. Dupixent is manufactured by Sanofi and Regeneron Pharmaceuticals, Inc. This is the first FDA-approved treatment for this condition. Learn more.

 

Research Opportunities

JOIN THE GLOBAL PAROXYSMAL NOCTURNAL HEMOGLOBINURIA PATIENT REGISTRY
AAMDS logo
Calling all paroxysmal nocturnal hemoglobinuria (PNH) patients and families! We need you to enroll in the Global PNH Patient Registry and share your experience. Help us advance research and development of standardized care.
 
ENROLL IN NORD'S METACHROMATIC LEUKODYSTROPHY HOME STUDY
NORD HOME Study graphic
Metachromatic Leukodystrophy research needs YOU! Get involved in progressing innovation by enrolling in NORD’s HOME Study and challenging others to do the same.
 
 

Rare Diseases Cures Accelerator - Data & Analytics Platform News

WORKSHOP RECORDINGS NOW AVAILABLE!
Thanks to all who joined us for the 2022 RCDA-DAP Workshop! Presentations are now available on demand on the Critical Path Institute YouTube page, including case studies and a discussion on the future of RCDA-DAP and the patient voice.
LEARN ABOUT DATA LITERACY
How do we get from medical research to making an improvement in people's lives? Check out the latest video for patients and caregivers from the RDCA-DAP education team on Data Literacy!
WATCH: THE ROLE OF INTEGRATED DATASETS
How can digitalization foster data collaboration and integrative analysis across various diseases? Watch the fifth installment of our RCDA-DAP 2022 Professional Webinar Series, “The Role of Integrated Datasets in Addressing Rare Diseases Research Challenges,” cohosted by GeneData, on demand now.
JOIN OUR WEBINAR ON DATA STEWARDSHIP
In catalyzing the gathering and organization of valuable patient data, it is essential to ensure integrity, security and privacy. Learn about best practices utilized by NORD and C-Path in the final RDCA-DAP webinar in our 2022 Professional Webinar Series, “Data Stewardship,” taking place on Wednesday, December 14.
SEE HOW RDCA-DAP CAN SUPPORT YOUR WORK
Hear from members of the RDCA-DAP team at upcoming conferences in November, including the International Congress for Ataxia Research, the AMIA 2022 Annual Symposium, the BioTechX and the World Orphan Drug Congress Europe 2022.
 

Member News

ACCESS BULLOUS DISEASE EDUCATION & SUPPORT

Connect with other pemphigus and pemphigoid patients and caregivers in the
Mid-Atlantic Area on Saturday, November 19. This is a free, virtual meeting.
JOIN THE CHARCOT-MARIE-TOOTH CLINICAL SUMMIT

The Charcot-Marie-Tooth Association will host the 2022 Clinical Summit for health professionals on Friday, November 4, with the goal of expanding their commitment to improving care for all people living with Charcot-Marie-Tooth disease. Multiple in-depth accredited sessions will be offered for medical teams including neurologists, physical therapists, occupational therapists, genetic counseling, and more!
EXPLORE THE CDMRP RARE OVARIAN CANCER NETWORKING PLATFORM

The Department of Defense’s Congressionally Directed Medical Research Programs (CDMRP) division is highlighting the Network for Rare Tumors of the Ovary (NRTO). The NRTO will serve as a central hub for disease information, research resources, and a connector out to two separate registries specific for rare ovarian tumor types.
IN-PERSON: JOIN THE CURE PSP FAMILY CONFERENCE

Join CurePSP for their 2022 Fall Family Conference! The conference is an opportunity to learn more about PSP, CBD, and MSA as well as develop a sense of community. The 2022 Fall Family Conference will take place at the Philadelphia Airport Marriott on Monday, November 14. CurePSP will partner with Penn Medicine to provide informative presentations on multidisciplinary care, physical therapy, research updates, social work, palliative care, and more!
VIRTUAL: JOIN THE CURE PSP FAMILY CONFERENCE

The CurePSP
Virtual Fall Family Conference will consist of pre-recorded sessions from the Philadelphia Fall Family Conference and live Q&A sessions. The first part of the virtual conference will be shown on Friday, December 9 and the second part will be shown on Friday, December 16. All sessions will be recorded and posted on the CurePSP YouTube channel after the event. Registration is free of charge!

ATTEND THE FDA CBER PATIENT-FOCUSED DRUG DEVELOPMENT MEETING

The FDA Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT) announced the Patient-Focused Drug Development listening meeting — Patient Perspectives on Gene Therapy Products on Tuesday, November 15. This meeting is an opportunity for patients, caregivers, patient advocates, and other important stakeholders to share their perspectives regarding gene therapy products, including cell-mediated gene therapies. Individuals interested in speaking at this public meeting can sign up during registration.
REGISTER FOR THE FOXG1 SYNDROME SCIENCE SYMPOSIUM & PARENTS CONFERENCE

FOXG1 Research Foundation is excited to announce that the first-ever FOXG1 Syndrome Science Symposium & Parents Conference will be held on November 4-6 at the Marriott Resort Fort Lauderdale Pompano Beach! The theme of the parents conference is Connect – Learn – Inspire. Parents will have the opportunity to meet scientists and clinicians, learn the FOXG1 Research Foundation's strategy towards a cure, and more.

MSUD FAMILY SUPPORT GROUP IS HIRING!

The MSUD Family Support Group is seeking a full-time executive director to manage day-to-day activities. This exciting new opportunity is a paid position.
ATTEND A MYASTHENIA GRAVIS COMMUNITY HEALTH FAIR NEAR YOU

The Myasthenia Gravis Foundation of America kicks off its first-ever local, in-person Community Health Fairs in 2022 to bring the myasthenia gravis community together in locations around the country. The goal is to effectively support people living with myasthenia and their caregivers, connect them with others to share stories and guidance, and provide education about managing myasthenia gravis. Join MGFA at an upcoming event in Atlanta, GA or Tampa Bay, FL.
ATTEND A NEUTROPENIA EDUCATIONAL SERIES

The Barth Syndrome Foundation and the National Neutropenia Network will host a neutropenia educational series over the next two months. Join the November events to learn about neutropenia from experts who will focus on the specific concerns of patients and parents about living with or raising a child who suffers from this lifelong condition.

REGISTER FOR THE PROGERIA INTERNATIONAL SCIENTIFIC WORKSHOP

The Progeria Research Foundation’s 11th International Scientific Workshop – RACE Progeria to the Cure will take place on November 2-4 in Boston, MA. Scientific workshops provide a platform that fosters collaboration among top minds in Progeria research, cardiovascular disease, and aging.

Join RACE to meet children and young adults with progeria and interact with the progeria basic and clinical scientific communities. Don’t miss cutting-edge scientific presentations, poster sessions, and high-level collaborative discussions that will lead to the next wave of treatments and a cure!
EXPLORE A SARCOIDOSIS RESEARCH CAMPAIGN AND EDUCATIONAL WEBINARS

The Foundation for Sarcoidosis Research (FSR) is launching the
Ignore No More: ACTe Now! (Advance Clinical Trials for Equity in Sarcoidosis) Campaign to address racial health disparities by increasing representation of Black sarcoidosis patients in clinical trials. FSR created a survey to provide a platform for Black Americans to share their experiences to help inform recommendations for improving care and increasing representation in clinical trials. The survey is available now through Wednesday, November 30! You can access the patient survey, FSR ACTe Now! Social Media Toolkit, and educational infographic on the campaign website to make sharing messaging easy on the FSR website. Learn more.

Join FSR on Saturday, November 12 for the Wellness Webinar: Navigation the Holidays with Sarcoidosis with Sandra Manley- Eichler, a Behavioral Health Integration Clinician. Sandra will share her tips to help patients manage sarcoidosis during the holidays. Register now.

FSR is thrilled to present its next Education Webinar: Skin Sarcoidosis on Wednesday, November 16. Dr. William Damsky, a physician-scientist who uses basis and translational approaches to understand mechanisms of skin disease, will join the event to discuss skin sarcoidosis. Register now.

Sarcoidosis can take a lot from patients and relationships. FSR’s next webinar is a Wellness Webinar: Sarcoidosis and Sexual Wellness. Jillian Bissar, a social worker and sexual health advocate will join FSR for this discussion on Tuesday, December 6. Register now.

Medications to treat sarcoidosis can often have negative side effects. Join FSR on Wednesday, December 14 for the Education Webinar: Managing Sarcoidosis and Treatment Related Side Effects. Three experts will share their treatments with attendees! Register now.
SUPPORT A TAY-SACHS & ALLIED DISEASES FUNDRAISER

Join the National Tay-Sachs & Allied Diseases (NTSAD) on Thursday, November 10 at the Royal Sonesta Boston for the Imagine & Believe 65th Anniversary Fundraiser where they envision a world with effective treatments for Tay-Sachs, Canavan, GM1, and Sandhoff diseases.

At Imagine & Believe, research pioneer Guangping Gao, PhD, will be honored for his life’s work in identifying the Canavan gene, revitalizing gene therapy, and his many research accomplishments. Imagine & Believe includes a reception, silent auction, and a speaking program. Each year the NTSAD Community supports families by raising funds for programs, services, and research.
 
Facebook
 
Twitter
 
Linkedin
 
Youtube
 
Instagram
 
Website
 
NORD logo
NORD, a 501(c)(3) organization, is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 330 patient organization members, is committed to the identification, treatment and cure of rare disorders through programs of education, advocacy, research and patient services. rarediseases.org
Click here to manage your preferences or unsubscribe completely.

National Organization for Rare Disorders, 1900 Crown Colony Drive, Suite 310, Quincy, MA 02169, United States


Email Marketing by ActiveCampaign