Paciente com tipo raro de esclerose mantém movimentos e segue trabalhando um ano após tratamento com medicamento 'personalizado'

A patient with a rare form of ALS showed stable or slightly improved condition after one year of treatment with a personalized drug targeting a specific genetic mutation. The RNA-targeting therapy, an antisense oligonucleotide, reduced damaging protein production, with biomarker and clinical tests indicating disease activity decline, supporting further research.