FDA approval of drug for rare genetic disorder gives families hope
Credit: OHSU News
Eric Stewart has a rare disease known as CTX. His mother, Susan Stewart, brought him to OHSU for his diagnosis and she continues to advocate for CTX screening and treatment. Researchers at OHSU discovered biomarkers essential to identifying and treating the disease, which led to a clinical trial at OHSU that in February culminated in FDA approval of an effective treatment for patients.