Rare Diseases
While Cures Within Reach is disease-agnostic, we are dedicated to improving the quality and length of life for patients living with a rare disease through the speed and cost-effectiveness of medical repurposing.
Our events and research within the Rare Diseases Repurposing Community all focus on connecting our many stakeholder groups together in our shared goal: driving more treatments to more patients more quickly through repurposing.
Open Funding Opportunity RFPs for Rare Diseases:
- Drug Repurposing Clinical Trials to Impact Blood Cancers: for clinical repurposing trials testing repurposed drugs in any blood cancer from anywhere in the world – accepting budgets of up to $75,000. CLOSED; THREE CLINICAL TRIALS SELECTED FOR FUNDING.
- CureAccelerator Live! for Diversity, Equity and Inclusion (DEI) in Rare Diseases: for clinical repurposing trials in any rare disease from US research institutions testing any repurposed therapy and that support the US-based Cures Within Reach DEI efforts for underrepresented racial / ethnic minority patients and researchers – accepting budgets of up to $70,000. CLOSED; THREE CLINICAL TRIALS SELECTED FOR FUNDING.
More information on our Funding Opportunities / RFPs page.
Events
Diversity of Clinical Trial Researchers and Patients: A Repurposing Opportunity?
November 2021
Virtual
A dialogue on the dual opportunities of clinical trial diversity to impact health disparities and/or improve minority leadership in scientific research.
Real World Data Through a Patient’s Lens
October 2021
Virtual
A conversation on Real World Data (RWD) and case reports from clinicians and researchers.
CureAccelerator Live! for Pediatric Rare Diseases 2021
June 2021
Virtual
Four finalists from institutions across the US and Chile competing for up to $50,000 to fund their repurposing clinical trials, all focused in pediatric rare diseases.
Repurposing for Pediatric Patients: Best Practices and Lessons Learned
November 2020
Virtual
A patient-focused conversation on the opportunities that repurposing drugs, devices and other treatments provide for the pediatric patient population.
How Patients Impact the 505(b)(2) Regulatory Process
October 2020
Virtual
A patient-focused conversation on the 505(b)(2) regulatory process and how patients, caregivers and patient advocacy groups can engage with both industry and regulators to support the drug development process, share their real-world perspectives for clinical trial planning and represent the patient view throughout the drug approval process.
Cure Accelerator Live! for Chicago 2020
September 2020
Virtual
Four finalists from the greater Chicago area competing for up to $50,000 to fund their repurposing clinical trials.
CureAccelerator Live! for Rare Diseases 2020
June 2020
Virtual
Five finalists from the US competing for up to $50,000 to fund their repurposing clinical trials impacting rare disease patients.
The Future of Patient Care in Acute Myeloid Leukemia (AML) and Other Blood Cancers
September 2019
Palo Alto, CA
A patient education event providing insight into current approaches to treating and living with AML and into potential future AML therapies.
CureAccelerator Live! for Rare Diseases 2019
June 2019
Philadelphia, PA
Three finalists from the US and Italy competing for up to $50,000 to fund their repurposing clinical trials impacting rare disease patients.
CureAccelerator Live! for Oncology 2017
November 2017
Washington, DC
Five finalists from institutions across the US competing for up to $50,000 to fund their oncology repurposing clinical trials.
Latest News
- CHOP Doctor Awarded the American Society for Bone and Mineral Research Frederic C. Bartter Award
- MYELOMA - S. Vincent Rajkumar, MD
- Vincent Rajkumar, M.D., Receives 2018 Distinguished Mayo Clinic Investigator Award
- ALZHEIMER’S TRIAL WILL “BATHE” PATIENTS’ BRAINS IN HELPFUL GENES
- Impact of Repurposing on Patients
- Opportunity That Clinical Repurposing Trials Brings to Patients
- How Your Donation to Cures Within Reach Creates Real Patient Impact
- Cures Within Reach Funds Huntington’s Disease Clinical Trial with Nilotinib at Georgetown
- Podcast: Impact Philanthropy Accelerating Cures Within Reach
Current Rare Research Projects
Completed Rare Research Projects
(in the past 3 years)
