Thanks to the generous financial support of our donors, Giving Strength is proud to fund groundbreaking research conducted by world-renowned scientists and clinicians focused on SELENON-RM. Current programs will lay the groundwork for discovering and developing new therapies for SELENON-RM and are strategically designed to achieve four critical goals:
A comprehensive understanding of the cellular and molecular underpinnings of SELENON-RM is essential for developing effective therapeutic interventions. By modeling disease processes using cells, animals, or computational approaches, we can reveal the underlying disease mechanisms and precisely target new treatments to the molecules and pathways requiring intervention. These models also serve as valuable tools for evaluating drug efficacy and safety.
Different diseases may share common molecular and cellular pathways. By identifying and repurposing drugs already tested for other conditions, we can significantly reduce both the time and cost of drug development, bringing effective treatments to patients more quickly and efficiently.
While SELENON-RM might share characteristics with other diseases, it is also a distinct condition caused by mutations in the SELENON gene, which encodes a protein with unique functions. Targeted therapies that address these specific genetic mutations, or correct the biological dysfunctions arising from the absence or impaired function of Selenoprotein N, may be necessary to effectively treat SELENON-RM.
Demonstrating a drug’s effectiveness in clinical trials is required for regulatory approval and marketing of the drug. To run a successful clinical trial, researchers need to identify desirable, measurable and achievable patient outcomes. Such outcomes might include how a patient feels based on a predefined scaling system, how a patient functions in a medical evaluation, or the level of a disease-associated biological or molecular marker (a biomarker). Predetermination of relevant clinical outcome measures allow medical professionals to objectively assess whether a drug provides value to patients. While every drug tested in a particular disease will require additional considerations to determine dose, duration of treatment and many other factors, the establishment of appropriate clinical outcome measures is a valuable step in preparing for testing any potential therapeutic.
