ICVS develops a new therapeutic mechanism for Machado–Joseph disease
Researchers from ICVS, in collaboration with i3S, University of Coimbra and international partners, have unveiled a promising new strategy for the treatment of Machado–Joseph disease, also known as Spinocerebellar Ataxia Type 3 (SCA3). The study, published in the prestigious journal Advanced Science, focuses on preventing the aggregation of mutant ataxin-3 protein, the molecular cause of this rare, inherited neurodegenerative disease, for which there is currently no cure.
