The FreeDOM-DM1 study of the antisense agent PGN-EDODM1 has its clinical hold lifted by FDA.

Published Date: 19 Oct 2023

Treatment with PGN-EDODM produced a greater than 68 percent correction of downstream transcript missplicing events in preclinical data by reducing pathogenic nuclear foci by 54% per nucleus and freeing muscleblind-like splicing regulator 1.

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