Article

A Phase 2 open-label study to determine the safety and efficacy of weekly dosing of ATL1102 in patients with non-ambulatory Duchenne muscular dystrophy

2022-01-19

Abstract excerpt

<h4>Background</h4> ATL1102 is a 2’MOE gapmer antisense oligonucleotide to the CD49d alpha subunit of VLA-4. ATL1102 inhibits expression of CD49d on lymphocytes, thereby reducing their survival, activation and migration to sites of inflammation. Children with Duchenne muscular dystrophy (DMD) have dystrophin deficient muscles. These are susceptible to contraction induced injury which triggers the immune system, ex...

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Literature Corpus work: 7fba7310-895d-520a-a112-0408f29d7bf9
DOI: 10.1101/2022.01.16.22269029

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