Preliminary results from the CONNECT1 clinical trial showed a favorable safety profile along with increased exon skipping and dystrophin production. DALLAS—PGN-EDO51, an exon 51 skipping therapy for ...

The company’s shares rose by about 5% in early trading. Although small, the Phase 2 trial showed that a so-called exon-skipping therapy can result in “substantial improvements” in muscle health, Wave ...

The drug is an oligonucleotide being studied in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping. The data, from 11 boys ages 5-11 with DMD—10 ambulatory and one ...

(RTTNews) - Entrada Therapeutics, Inc. (TRDA), on Monday announced that it has received authorization from the United Kingdom's Medicines and Healthcare Products Regulatory Agency or MHRA and ...

Exon skipping is based on instructing cells to "skip" these mutations during protein production, with the hope of restoring a functional protein. In 2024, the PMO franchise earned $967m of ...

Avid­i­ty Bio­sciences shared Mon­day that its RNA drug for peo­ple with cer­tain mu­ta­tions of Duchenne mus­cu­lar dy­s­tro­phy con­tin­ued to show strong re­sults as it com ...

Michelle Obama on Tuesday appeared to open up publicly for the first time about her decision to skip President Trump’s inauguration, saying she’s at the point in her life where she’s ...

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