ISS-N1 makes the first FDA-approved drug for spinal muscular atrophy

ISS-N1 makes the first FDA-approved drug for spinal muscular atrophy

Spinal muscular atrophy (SMA) is one of the leading genetic diseases of children and infants. SMA is caused by deletions or mutations of Survival Motor Neuron 1 (SMN1) gene. SMN2, a nearly identical copy of SMN1, cannot compensate for the loss of SMN1 due to predominant skipping of exon 7. While var...

Full description

Bibliographic Details
Main Author: Ottesen Eric W.
Format: Article
Language:English
Published: De Gruyter 2017-01-01
Series:Translational Neuroscience
Subjects:
spinal muscular atrophy
antisense oligonucleotides
survival motor neuron protein
smn
spinraza
isis-smnrx
ionis-smnrx
nusinersen
iss-n1
splicing regulation
Online Access:https://doi.org/10.1515/tnsci-2017-0001

Internet

https://doi.org/10.1515/tnsci-2017-0001

Similar Items