Initial results from FREEDOM-DM1 trial demonstrate significant splicing correction of 29.1% at 10 mg/kg dose in patients with Myotonic Dystrophy Type 1, showing potential therapeutic efficacy.
The myotonic dystrophy therapeutic landscape is experiencing a surge in R&D, with over 20 companies actively involved in developing 22+ novel therapies.
PepGen's PGN-EDO51 Phase 2 study shows promising initial data with mean exon skipping in biceps tissue of 2.15% at week 13 for Duchenne muscular dystrophy.
PepGen anticipates releasing preliminary data from its Phase II CONNECT1-EDO51 trial of PGN-EDO51 in mid-2024, offering a potential treatment for Duchenne muscular dystrophy (DMD).
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