Explore a past NAF research grant awardee's funded study, gaining scientific insights about their Ataxia research.
Dr. Magda Santana presented the research, "A systems approach to find a blood-based biomarker for Machado-Joseph Disease." on December 15th, 2023.
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.
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Speaker:
Magda M Santana PharmD, PhD is a junior researcher interested in translational research in the Ataxia field. As undergraduate student, MMS was in the ranking of the 3% Best Students of Pharmaceutical Sciences. MMS finished her Integrated Master in Pharmaceutical Sciences (final classification 17/20), in 2008, and got her Doctoral degree in Pharmacy (Pharmacology and Pharmacotherapy) at University of Coimbra, Portugal, in 2013. In 2014, Magda was invited by Prof. Luis Pereira de Almeida to join the Gene and Stem Cell Therapies for the Brain Group at Center for Neuroscience and Cell Biology from the University of Coimbra, Portugal. Since then, she has been focusing her research on the development of biomarkers, pre-clinical models (iPSCs and rodents) and drug/gene therapies for MJD. In total, Magda has been part of the research team of over 25 projects and has received financial support to develop 3 projects as Principal investigator in ataxia field. Magda performed important pre-clinical studies with trehalose for the treatment of spinocerebellar ataxia type 3 which contributed to the initiation of a clinical trial in SCA3. Moreover, Magda have been playing a key role in ESMI project, being coordinator of the ESMI biobank and highly contributing to advances in the development of new biomarkers for SCA3. Magda is an active member of the SCA Global and SCA Global Young Investigator Initiative and has also engaged in several scientific communication and public activities particularly dedicated to SCA3 patients and their families, in collaboration with the Portuguese Association for Hereditary Ataxias.
16 окт 2024
