FDA approves first drug for spinal muscular atrophy | FDA
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 strength and ...
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 treatment of spinal...
The Safety and Efficacy of Nusinersen in the Treatment of Spinal ...
Background and objectives: Spinal muscular atrophy (SMA) is a neurodegenerative disease that leads to progressive proximal muscle weakness and muscle atrophy. To assess the beneficial and adverse effects of nusinersen, a promising intervention for ...
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 wasting. The most common pathology results from a homozygous disruption in the survival motor neuron 1 (SMN1) gene on chromosome 5q13 via deletion, conversion, or mutation.
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.
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.
FDA Accepts sNDA for Higher Dosing Regimen of Nusinersen for Spinal …
6 days ago · According to a new announcement, the FDA has accepted Biogen’s supplemental new drug application (sNDA) for a higher, potentially more efficacious dose regimen of nusinersen (Spinraza) for patients with spinal muscular atrophy (SMA).
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 SMA in children and adults.
Nusinersen for Spinal Muscular Atrophy in the United States ... - PubMed
Nusinersen, an antisense oligonucleotide that increases expression of the functional SMN protein, was approved for SMA by US and European regulatory agencies in 2016 and 2017, respectively. The indicated regimen requires intrathecal injections every 4 months, following the first four injections during the loading phase.
FDA accepts sNDA for higher regimen nusinersen for spinal muscular atrophy
5 days ago · The FDA has accepted a supplemental New Drug Application for a higher dose regimen of nusinersen (Spinraza; Biogen Inc) for spinal muscular atrophy (SMA), according to a recent announcement from Biogen. 1 The higher dose is made up of a more rapid loading regimen, two 50 mg doses at 14 days apart, and higher maintenance regimen, 28 mg every 4 months, compared to the the currently approved ...