Eteplirsen therapy for Duchenne muscular dystrophy: skipping to the front of the line

Nat Rev Neurol. 2016 Dec;12(12):675-676. doi: 10.1038/nrneurol.2016.180. Epub 2016 Nov 18.

Author

James J Dowling¹

Affiliation

¹ Division of Neurology and Program for Genetics and Genome Biology, Hospital for Sick Children, 555 University Avenue, Toronto, Ontario, Canada, M5G 1X8.

PMID: 27857122
DOI: 10.1038/nrneurol.2016.180

No abstract available

MeSH terms

Adolescent
Child
Clinical Trials as Topic
Gene Deletion
Humans
Male
Morpholinos / administration & dosage
Morpholinos / pharmacology*
Muscular Dystrophy, Duchenne / drug therapy*
Oligonucleotides, Antisense*

Substances

Morpholinos
Oligonucleotides, Antisense
eteplirsen