Resultados prometedores para el primer paciente con esclerosis lateral amiotrófica tratado con una terapia de ARN.

N-of-1 investigational study of a novel antisense oligonucleotide drug in CHCHD10-related ALS shows early signs of efficacy Margot A. Cousin1,2,3 ∙ Aditi I. Dagli4 ∙ Laurence Mignon5 ∙ … ∙ Zachary T. McEachin11,12 ∙ Leonard Petrucelli7,13 ∙ Björn Oskarsson https://www.cell.com/med/abstract/S2666-6340(26)00298-9?_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS2666634026002989%3Fshowall%3Dtrue&utm_medium=email&_hsenc=p2ANqtz--DYpETI-W0TMJR7CeIdZIeIZst-YnC2_0cNz6wAB0pq-ADi4zielAXGbxzM6FmQrsidyGbdg0NsCnOEpdQdQQ7UqLKTg&_hsmi=146512528&utm_content=146523058&utm_source=hs_email%3D