RARE Science Goals for our research and clinical programs:
- Empower Patient families/Foundations with tools to “own” their data and unite patient communities which focus on bringing together a critical amount of data enabling biological understanding of the disease driving toward therapeutic solutions.
- Accelerate identification of more immediate therapeutic solutions for kids with rare disease potentially through repurposing currently approved drugs and other therapeutic approaches.
- Provide tools, routes of communication and strategy that capture information that can be acted on in an efficient way across all stakeholders (patient family, Researcher, Clinician) addressing the urgency needed to find solutions.
- Provide tools through the RARE Hub which is a neutral data agnostic common computational platform that links all stakeholders – the patient family/foundation, research and clinical to close the gap of time between discovery and actual clinical application.
- Through our partner Agility clinical, a recognized leader in rare disease clinical strategy provide clinical strategy and resources to conduct international rare disease trials. Agility Clinical is the first orphan drug focused clinical trial services organization in the US. Due to experience and innovation in small trial design the organization has become known for successful Orphan/Rare clinical trial design and execution.
Common computational platform can integrate all stakeholders and close the gap of time between discovery and clinical treatments
