Science Showcase: Dr. Santana
A Systems Approach to Find a Blood-Based Biomarker for Machado-Joseph Disease – presented by Dr. Magda Santana.
Science Showcase sessions explore a past NAF research grant awardee’s funded study to gain scientific insights about their Ataxia research.
Register for Webinar: Click here.
Lay Summary: In this project, we aim at identifying and developing a blood-based disease progression biomarker for MJD, by combining data obtained from analysis of proteins and RNA in biological fluids and patient’s neuronal cells derived from induced pluripotent stem cells, with brain imaging and clinical data of a large longitudinal cohort of MJD patient’s. The development of a biomarker for MJD will allow the development of a clinical trial using a biomarker as an endpoint. So, this project will have a remarkable impact in the field contributing to accelerate the discovery of an effective therapy for MJD. This project will also have a direct impact on health care as the biomarker can be used by clinicians in the monitoring and clinical management of patients.