Global Genes is pleased to announce the rare disease patient advocacy organizations receiving 2024 RARE Research Readiness Fellowships. As an exclusive funding opportunity for rare patient organizations that are a part of the Global Advocacy Alliance, this fellowship provides patient advocacy leaders with the opportunity to make a tangible difference in the lives of rare disease patients and caregivers across the globe.
The RARE Research Readiness Fellowship will provide awardees with high-touch support and mentorship around research readiness and strategic planning. The program will include a gap analysis of ongoing and completed research and assistance in developing a prioritization strategy. This will help guide awardees in making impactful decisions regarding their grant awards and community engagement activities.
Over 30 organizations submitted applications in the inaugural year for this funding program. Congratulations to the Global Advocacy Alliance members who received the 2024 RARE Research Readiness Fellowships!
CureARS
The fellowship will support CureARS’s efforts to create a strategic unifying research plan to understand the phenotypic presentations of the family of mtARS disorders. They can then efficiently utilize resources and uncover commonalities that siloed studies have previously missed. This strategic plan will connect researchers and patient advocacy groups while also formulating a comprehensive picture for the landscape for mtARS research, and filling in the gaps for what areas need to be further explored.
KARES Foundation
KARES Foundation will utilize its fellowship to help create a research plan that will build on already established partnerships with RARE-X, CombinedBrain, and NORD. The organization can then begin to transition from current preclinical research to research that will directly improve the quality of life for individuals affected by KDM5C genetic variants. Because KDM5C is an ultra-rare condition with only a few hundred cases globally, very few researchers are investigating the KDM5C gene in humans. KARES Foundation hopes that this fellowship will accelerate research in the condition.
MED13L Foundation
The fellowship that MED13L Foundation received will assist the organization in overcoming several hurdles that they have encountered recently after developing a strategic research plan. The challenges included a lack of infrastructure to engage the patient community to collect data and a natural history study. The fellowship will also allow the foundation to expand its scientific advisory board.
The Rory Belle Foundation
The Rory Belle Foundation will use the fellowship to help guide research funding decisions for the NARS1 community, while the newly created strategic research plan will help to establish a Medical and Scientific Advisory Board to provide guidance with clear priorities. The fellowship will complement existing research efforts like the established RARE-X registry.
Thank you to all of the Global Advocacy Alliance members who applied for a RARE Research Readiness Fellowship this year. For more information about supporting the RARE Impact Grants, contact Kristin.Soto@globalgenes or [email protected] (Business Development) or Global Advocacy Alliance. A special thank you to the sponsors of the 2024 RARE Research Readiness Fellowship: Amgen, Beam, and Travere.
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