Muscle microRNAs in the cerebrospinal fluid predict clinical response to nusinersen therapy in type II and type III spinal muscular atrophy patients
I Magen, S Aharoni, NS Yacovzada… - European journal of …, 2022 - Wiley Online Library
Background and purpose The antisense oligonucleotide nusinersen (Spinraza) regulates
splicing of the survival motor neuron 2 (SMN2) messenger RNA to increase SMN protein
expression. Nusinersen has improved ventilator‐free survival and motor function outcomes
in infantile onset forms of spinal muscular atrophy (SMA), treated early in the course of the
disease. However, the response in later onset forms of SMA is highly variable and
dependent on symptom severity and disease duration at treatment initiation. Therefore, we …
splicing of the survival motor neuron 2 (SMN2) messenger RNA to increase SMN protein
expression. Nusinersen has improved ventilator‐free survival and motor function outcomes
in infantile onset forms of spinal muscular atrophy (SMA), treated early in the course of the
disease. However, the response in later onset forms of SMA is highly variable and
dependent on symptom severity and disease duration at treatment initiation. Therefore, we …
P. 53 Muscle microRNAs in the cerebrospinal fluid predict clinical response to nusinersen therapy in type II and type III spinal muscular atrophy patients
The antisense oligonucleotide nusinersen (spinraza) regulates splicing of the survival motor
neuron 2 (SMN2) messenger RNA to increase SMN protein expression and has improved
ventilator free survival and motor function outcomes in infantile onset forms of SMA, treated
early in the course of the disease. However, the response in later onset forms of SMA is
highly variable and dependent on symptom severity and disease duration at treatment
initiation. Therefore, we aimed to identify novel noninvasive biomarkers that could predict …
neuron 2 (SMN2) messenger RNA to increase SMN protein expression and has improved
ventilator free survival and motor function outcomes in infantile onset forms of SMA, treated
early in the course of the disease. However, the response in later onset forms of SMA is
highly variable and dependent on symptom severity and disease duration at treatment
initiation. Therefore, we aimed to identify novel noninvasive biomarkers that could predict …
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