Rare Disease – November 2022 eNews

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).

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!

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.

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!

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.

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.

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.