Article

<i>Mdx</i> mice dosed with antisense to CD49d and dystrophin exon skip morpholino; reduced force loss and affected gene expression pathways support ATL1102 combination therapy in DMD patients

2024-11-24

Abstract excerpt

<h4>Background</h4> VLA-4 is a disease progression biomarker in patients with Duchenne Muscular Dystrophy (DMD) including those treated with corticosteroids. ATL1102 an antisense oligonucleotide (ASO) inhibitor of human CD49d chain of adhesion molecule VLA-4 shows promising phase-2 results in non-ambulant DMD stabilizing upper limb function (PUL2.0), grip strength, and MRI muscle fat fraction, compared to worseni...

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Literature Corpus work: 448aeac6-766b-5f13-86f5-71fc0a96836a
DOI: 10.1101/2024.11.23.625028

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