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Entering the era of precision medicine to treat amyotrophic lateral sclerosis

Abstract With the disease modifying therapy Qalsody (tofersen) which targets the RNA product of the SOD1 gene, having been shown effective in amyotrophic lateral sclerosis (ALS), the present perspective seeks to explore progress towards the implementation of precision medicine principles in ALS drug...

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Principais autores: Frances Theunissen, Loren Flynn, Alfredo Iacoangeli, Ahmad Al Khleifat, Ammar Al-Chalabi, James J. Giordano, Masha Strømme, P. Anthony Akkari
Format: Artigo
Sprog:Inglês
Udgivet: BMC 2025-10-01
Serier:Molecular Neurodegeneration
Fag:
Personalised medicine
Antisense oligonucleotide
Genetics
Understanding heterogeneity
Long-read sequencing
Patient profiling
Online adgang:https://doi.org/10.1186/s13024-025-00890-5
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