News Related to Composite Mis-splicing Score

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FDA Pauses U.S. Enrollment in Myotonic Dystrophy Trial After Serious Adverse Event

Mar 06, 2026

The FDA has placed a partial hold on U.S. enrollment in a Phase 2 trial of del-desiran for myotonic dystrophy type 1, citing preclinical mouse data showing blood pressure drops not seen in humans, following a serious adverse event in earlier testing.

Del-desiran Shows Muscle Delivery and Splicing Improvements in Phase 1/2 DM1 Trial

Feb 19, 2026

Phase 1/2 MARINA trial results published in NEJM show del-desiran achieved approximately 40% reduction in toxic DMPK mRNA in muscle tissue and improvements in myotonia and functional measures in myotonic dystrophy type 1 patients, with acceptable safety profile.