Identify and Define Patient Population: The first step to a cure, is to identify patients living with an ARS gene mutation. Researchers need to understand how the disease effects these patients now and over time.
Create disease models of ARS Genes: To better understand ARS genes, researchers create models of the genes in animals or using patient cells. These models include mice, zebrafish, worms, and iPSCs.
Study disease models of ARS Genes: Researchers can study disease models to better understand how ARS genes effect the body and how to treat these conditions.
Test potential treatments: Potential treatments can be tested in ARS disease models prior to giving the treatment to a patient. These treatments can include new treatments, repurposed drugs, or potential cures, such as a gene therapy. Before treating a patient with a potential therapy, researchers are required to prove a drug first works in an ARS disease model.
Involve Patients: Patient involvement is critical to advance research. Due to a small patient population, every patient involved is vital to our success. Opportunities to join patient registries, biosample donations, clinical trial involvement, or participation in data collection may be available.