February 28, 2019
The National Institutes of Health will host Rare Disease Day at NIH and open its doors to public and private researchers, patients and their support organizations, policy makers and other members of the community. The event will run from 8:30 a.m. to 4 p.m. ET on the NIH Campus, Natcher Conference Center (Building 45), Main Auditorium
45 Center Drive, Bethesda, Maryland. Together, attendees will raise awareness about rare diseases, the people they affect, and opportunities for new and better treatments. New features for this year’s event include a presentation of the first ever Zebbie Award for the Rare Diseases Are Not Rare! Challenge, sponsored by the National Center for Advancing Translational Sciences (NCATS). Also featured will be personal video stories by rare disease patients, video messages from global rare disease leaders and a special rare disease portrait unveiling by Beyond the Diagnosis of an NIH Clinical Center patient. The NIH Clinical Center — the nation’s largest research hospital where scientists are studying more than 500 rare diseases in partnership with nearly 2,250 patients — will also offer tours. Panel discussion topics include success factors for rare cancer research, collective research models with the NIH Rare Diseases Clinical Research Network, quality patient registries to understand rare diseases and gene editing/genome therapy clinical trials. For more information, visit the event page.


February 28, 2019

Proposals for repurposing research for rare disease therapies are due to Cures Within Reach. The organization recently launched the new funding opportunity for clinical repurposing research in any rare disease. This RFP will support clinical research that repurposes approved and available drugs, devices, nutraceuticals, and diagnostics.
Up to five submissions will be selected to pitch at the CureAccelerator Live! event, to be held in Spring/Summer 2019 where event attendees will select the winning project. The winning project can receive up to $50,000 in funding. Proposal submissions are due by February 28, 2019, on CureAccelerator. Full RFP details can viewed on the organization’s funding opportunities page.

March 15
The deadline for applications for the Shire ACES Scholarship program are due. The program awards scholarships of $5,000 for rare disease patients who graduate high school graduates or obtain a GED credential by July 2019, or are a current undergraduate college student. For details on eligibility or to learn more and apply, go to the ShireACES website.

April 10-12
The Global Orphan Drug Conference USA 2019 will be held April 10 to April 12 at the Gaylord National Harbor Hotel in Oxon Hill, Maryland. The World Orphan Drug Congress USA focusses on the most pressing challenges and opportunities to bring rare disease therapies to patients faster. It brings together 1,200 leaders in orphan drugs from 50 countries and will feature more than 135 presentations covering all aspects of orphan drug development and rare disease research. For more information and to register, go to the event page.

June 21-23
Living Rare, Living Stronger
NORD will bring the rare disease community together June 21 to June 23 in Houston, Texas for the 2019 Living Rare, Living Stronger NORD Patient & Family Forum. During this event, the rare community will come together with physicians, medical students, and allied health professionals for a program of learning, sharing and connection – in a fun atmosphere of support and understanding. For more information, visit the event webpage.

March 9
Uplifting Athletes will holds its 2019 Young Investigator Draft Saturday, March 9 at Lincoln Financial Field (home of the NFL’s Philadelphia Eagles). This unique one-of-a-kind event is a celebration that ties the theme of the NFL Draft and Rare Disease Research together, and is the result of Uplifting Athletes’ ongoing commitment to cultivate resources that accelerate scientific advancements for rare disease treatments and potential cures. This unique one-of-a-kind event is a celebration that ties the theme of the NFL Draft and Rare Disease Research together, and is the result of Uplifting Athletes’ ongoing commitment to cultivate resources that accelerate scientific advancements for rare disease treatments and potential cures. At the event, individual grants will be given to five Young Investigators who pursue rare disease research in these five different areas: rare cancers, rare autoimmune and immunological disorders, rare blood disorders, rare genetic disorders and rare muscular and neurological disorders. For more information or to register, go to the event page.

March 27-29
The Wellcome Genome Campus will hold a Genomics of Rare Disease meeting March 27 to 29  on the Wellcome Genome Campus, U.K. It is the 13th in a series of meetings on rare diseases, which will present a blend of genomic science and clinical medicine. This meeting will explore how cutting-edge genomic research translates into clinical care and informs our understanding of the biology of rare disease. For more information or to register, go to the event page.

May 7-9
The Professional Patient Advocates in Life Sciences (PPALS) Patient Advocacy Certificate Training will take place in Sioux Falls, South Dakota May 7-9. The training includes a patient advocacy professionals in industry track and a non-profit health advocacy leaders track. More information can be found on the event page.

May 8-11
The International Congress of Research on Rare and Orphan Diseases will hold the Re(Act) Congress America in Toronto May 8 to 11. The Congress aims to bring together scientific leaders and experts, and young scientists from a variety of breakthrough scientific fields to present cutting-edge research, and exchange ideas. Patients and patient organizations, who are committed to research, will also be in attendance to share their experiences and perspectives. For more information or to register, go to the event page.

September 18-19
Rally for Medical Research Hill Day will be held on September 18 to 19. Held every September, this event includes more than 300 national organizations coming together in support of the Rally for Medical Research. The purpose of the Rally is to call on our nation’s policymakers to make funding for National Institutes of Health a national priority and raise awareness about the importance of continued investment in medical research that leads to more progress, more hope, and more lives saved. The program includes a training session and reception on September 18 and a kick-off breakfast and meetings with House and Senate offices on September 19. For more information, go to the event page.

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This page was last updated October 29, 2018