Spinraza-a rare disease success story
Gene Ther
.
2017 Sep;24(9):497.
doi: 10.1038/gt.2017.59.
Author
V Prakash
1
Affiliation
1
Springer Nature Gene Therapy, London, UK.
PMID:
28963567
DOI:
10.1038/gt.2017.59
No abstract available
Publication types
Editorial
MeSH terms
Genetic Therapy / methods
Humans
Muscular Atrophy, Spinal / drug therapy*
Oligonucleotides / pharmacology*
Rare Diseases
Substances
Oligonucleotides
nusinersen