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A phase 2 open-label study of the safety and efficacy of weekly dosing of ATL1102 in patients with n...

A phase 2 open-label study of the safety and efficacy of weekly dosing of ATL1102 in patients with n...

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_plos_journals_3069269495

A phase 2 open-label study of the safety and efficacy of weekly dosing of ATL1102 in patients with non-ambulatory Duchenne muscular dystrophy and pharmacology in mdx mice

About this item

Full title

A phase 2 open-label study of the safety and efficacy of weekly dosing of ATL1102 in patients with non-ambulatory Duchenne muscular dystrophy and pharmacology in mdx mice

Publisher

United States: Public Library of Science

Journal title

PloS one, 2024-01, Vol.19 (1), p.e0294847-e0294847

Language

English

Formats

Publication information

Publisher

United States: Public Library of Science

More information

Scope and Contents

Contents

ATL1102 is a 2'MOE gapmer antisense oligonucleotide to the CD49d alpha subunit of VLA-4, inhibiting expression of CD49d on lymphocytes, reducing survival, activation and migration to sites of inflammation. Children with DMD have dystrophin deficient muscles susceptible to contraction induced injury, which triggers the immune system, exacerbating mu...

Alternative Titles

Full title

A phase 2 open-label study of the safety and efficacy of weekly dosing of ATL1102 in patients with non-ambulatory Duchenne muscular dystrophy and pharmacology in mdx mice

Identifiers

Primary Identifiers

Record Identifier

TN_cdi_plos_journals_3069269495

Permalink

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_plos_journals_3069269495

Other Identifiers

ISSN

1932-6203

E-ISSN

1932-6203

DOI

10.1371/journal.pone.0294847

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