Although not experimentally demonstrated, the authors in both publications suggested that the exonic mutations were responsible for the exon skipping.

An exon-skipping technique using dual single-guide RNA/Cas9 ribonucleoproteins targeted at 3 novel COL7A1 exons with pathogenic heterozygous mutations achieved exon deletion rates of up to 95%.

Additionally, data showed AOC 1044 was well-tolerated, demonstrated “statistically significant” exon 44 skipping compared with placebo of up to 1.5% in healthy volunteers after a single dose ...

This comprehensive comparative analysis examined the economic and health care resource utilization implications of initiating glucocorticoid and exon-skipping therapy for Duchenne muscular ...

Sarepta Therapeutics obtained traditional FDA approval for ambulatory DMD patients and accelerated approval for ...

The FDA has granted fast track designation to AOC 1044 for the treatment of Duchenne muscular dystrophy in people with mutations amenable to exon 44 skipping, Avidity Biosciences announced in a ...

SRP-5051 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping.

- Preliminary data from the 5 mg/kg PGN-EDO51 dose level in the CONNECT1-EDO51 Phase 2 trial are expected mid-2024: including initial safety, exon 51 skipping and dystrophin protein production ...

Vebreltinib for Advanced Non–Small Cell Lung Cancer Harboring c-Met Exon 14 Skipping Mutation: A Multicenter, Single-Arm, Phase II KUNPENG Study The following represents disclosure information ...

Part A is a multiple ascending dose study designed to evaluate the safety, pharmacokinetics and pharmacodynamics, including exon skipping and dystrophin production in approximately 24 patients.