ALS: first cure for patients with specific mutations

(ANSA) - TURIN, 22 SEPTEMBER - A slowing and in some cases a reversal of the clinical progression of myotrophic lateral sclerosis (ALS) have been found in people carrying the mutation in the Sod1 gene through the use of Tofersen, an antisense oligonucleotide (Aso) that acts selectively on messenger RNA, blocking the synthesis of the altered protein.

The therapy is administered by lumbar puncture, and this method is very well tolerated.

    The data of the international study, in which the Molinette hospital in Turin was involved in Italy, were published today in the prestigious New England Journal of Medicine.

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