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December 03, 2025 08:00 ET | Source: Arrakis Therapeutics

Arrakis Therapeutics Presents Data Highlighting Progress of Oral RNA-targeted Small Molecule to Treat Myotonic Dystrophy Type 1 (DM1)

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...