whole body delivery

Early results show that when AVI4658 is delivered system-wide through the bloodstreams of boys with DMD, it’s safe and increases dystrophin production.

posted on December 23, 2009 - 3:07pm
Interim results from a human clinical trial of the exon-skipping compound AVI4658 in boys with Duchenne muscular dystrophy (DMD) show that when the compound is delivered to the whole body via the bloodstream — rather than simply injected into a foot muscle as in a previous trial — it appears safe and leads to production of the missing muscle protein dystrophin.

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