Contrato para el desarrollo del proyecto Estudio ESMI - European Spinocerebellar Ataxia type3/Machado-Joseph Disease lnitiative
Datos básicos
- Código del Financiador:
- CSI24/06
- Año Inicial:
- 2024
- Año final:
- 2026
Objetivos del proyecto
The aim of this study is to compile a trial-ready cohort of sufficient size consisting of patients with spinocerebellar ataxia type 3 (SCA 3) and first-degree relatives, so-called risk persons, for future drug studies. Spinocerebellar ataxia type 3 (SCA 3) is also known as Machado-Joseph's disease, the most common hereditary ataxia. The causal gene mutation is known, but there is no therapy available yet. A main focus of the study is to identify biomarkers that show the disease severity at a very early stage. These are of particular importance for future drug studies as developmental parameters.
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Participantes
Unidades de investigación
Financiadores
UCB BIOPHARMA SRL