Wave clinical trial for Duchenne amenable to skipping exon 53 currently enrolling in US

exon 53

Wave Life Sciences has announced that they are initiating Part C of the FORWARD-53 trial—an open-label Phase 1b/2 study of up to 15 ambulatory individuals aged 4-10 years and amenable to skipping exon 53.  Participants will receive an IV infusion of WVE-N531 every 4 weeks following an initial loading dose regimen, and will undergo muscle biopsies before their first dose and following 24 weeks of treatment.    Four US sites are currently screening participants. 

More details can be found in a letter to the community from Wave.

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