Anti-amyloid treatment is broadly effective in neuronopathic mucopolysaccharidoses and synergizes with gene therapy in MPS-IIIA.
Giaccio M, Monaco A, Galiano L, Parente A, Borzacchiello L, Rubino R, Klärner FG, Killa D, Perna C, Piccolo P, Marotta M, Pan X, Khijniak M, Siddique I, Schrader T, Pshezhetsky AV, Sorrentino NC, Bitan G, Fraldi A.
Giaccio M, et al. Among authors: schrader t.
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Mol Ther. 2024.
PMID: 39342429
Free PMC article.