Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
Feb 14, 2018 · Nusinersen is an antisense oligonucleotide drug that modulates pre–messenger RNA splicing of the survival motor neuron 2 (SMN2) gene. It has been developed for the …
Nusinersen: A Treatment for Spinal Muscular Atrophy - PubMed
It is a novel modified antisense oligonucleotide designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron protein deficiency. Nusinersen has been …
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
Nov 2, 2017 · We conducted a randomized, double-blind, sham-controlled, phase 3 efficacy and safety trial of nusinersen in infants with spinal muscular atrophy.
Long-term efficacy and safety of nusinersen in adults with 5q spinal ...
Oct 24, 2023 · Evidence for the efficacy of nusinersen in adults with 5q-associated spinal muscular atrophy (SMA) has been demonstrated up to a period of 16 months in relatively large …
Nusinersen for spinal muscular atrophy - PMC - PubMed …
However, the recently approved antisense oligonucleotide (ASO) nusinersen now provides not only an upcoming and promising treatment option for SMA, but may also represent a general …
Nusinersen: A Novel Antisense Oligonucleotide for the Treatment …
Nusinersen, an antisense oligonucleotide, is administered directly into cerebrospinal fluid. It alters SMN2 pre-RNA splicing so exon 7 is included, increasing expression of functional SMN protein.
Long-term nusinersen treatment across a wide spectrum of spinal ...
Aug 4, 2023 · Spinal muscular atrophy (SMA) is an autosomal recessive disorder caused by a biallelic mutation in the SMN1 gene, resulting in progressive muscle weakness and atrophy. …
Nusinersen (Spinraza) for Spinal Muscular Atrophy Treatment
One of the latest breakthroughs in care for patients with spinal muscular atrophy (SMA) is the FDA approval of a new medicine called nusinersen (brand name Spinraza) for the treatment of …
Nusinersen for the treatment of spinal muscular atrophy
Spinal muscular atrophy (SMA) is an autosomal recessive degenerative neuromuscular disorder characterized by loss of spinal motor neurons leading to muscle weakness. This review article …
Nusinersen as a Therapeutic Agent for Spinal Muscular Atrophy
Nusinersen is the first new oligonucleotide-based drug targeting the central nervous system for the treatment of SMA. This review of nusinersen will discuss its action mechanism, cellular …