Rare Disease Report

New Antisense Therapy in Development for Pompe Disease

FEBRUARY 12, 2015

In this exclusive interview during WORLDSymposium 2015, Nicholas Clayton, MSc, staff scientist at Genzyme Corporation discusses the exciting new data he and his colleagues presented on antisense therapy that is in development to treat patients with Pompe disease.

Clayton N, et al. Antisense oligonucleotide‚Äźmediated suppression of muscle glycogen synthase 1 synthesis as an approach for substrate reduction therapy of Pompe disease. Poster presented at: WORLDSymposium 2015; Orlando, Fla; February 10-12, 2015.

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