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— Mark DeLong to lead Arrakis as it becomes a clinical-stage company, advancing its lead oral RNA-targeted small molecule to treat myotonic dystrophy type 1 (DM1) — — Michael Gilman retires from role...
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Preclinical in vivo data demonstrate hyper-precise mechanism directly targets the disease-causing mRNA repeats leading to correction of splicing defects and reversal of myotonia Oral administration...