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 for spinal muscular atrophy - PubMed Central (PMC)
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 (Spinraza®) – Spinal Muscular Atrophy (SMA)
Nusinersen, marketed in the U.S. as Spinraza ® (Biogen) is the first therapy approved for the treatment of SMA. SMA results from mutations in a gene known as SMN1, which encodes a …
Nusinersen for the treatment of spinal muscular atrophy
This review article focuses on a novel antisense oligonucleotide treatment, first ever approved for SMA (nusinersen, Spinraza TM) and describes the exciting journey from early ASO clinical …
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: 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.
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 …
FDA approves first drug for spinal muscular atrophy - U.S. Food …
FDA approved Spinraza (nusinersen), the first drug approved to treat children and adults with spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle …
The Antisense Oligonucleotide Nusinersen for Treatment of Spinal ...
Spinal muscular atrophy (SMA) is a rare, autosomal recessive neuromuscular degenerative disease characterized by loss of spinal cord motor neurons leading to progressive muscle …
FDA accepts sNDA for higher regimen nusinersen for spinal muscular atrophy
5 days ago · Nusinersen is an injection that is administered into the fluid surrounding the spinal cord.2 “Continued progress to improve upon the remarkable initial successes in SMA …