Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study
Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study
About this item
Full title
Author / Creator
Publisher
United States: John Wiley & Sons, Inc
Journal title
Language
English
Formats
Publication information
Publisher
United States: John Wiley & Sons, Inc
Subjects
More information
Scope and Contents
Contents
Objective
The novel morpholino antisense oligonucleotide viltolarsen targets exon 53 of the dystrophin gene, and could be an effective treatment for patients with Duchenne muscular dystrophy (DMD). We investigated viltolarsen’s ability to induce dystrophin expression and examined its safety in DMD patients.
Methods
In this open‐label, mult...
Alternative Titles
Full title
Viltolarsen in Japanese Duchenne muscular dystrophy patients: A phase 1/2 study
Authors, Artists and Contributors
Identifiers
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Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_7732240
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_7732240
Other Identifiers
ISSN
2328-9503
E-ISSN
2328-9503
DOI
10.1002/acn3.51235
