A new article argues dystrophin immunogenicity is an under-examined issue in the treatment of Duchenne muscular dystrophy.

Wave Life Sciences’ Phase II open-label trial investigating its disease-modifying drug for boys living with Duchenne Muscular ...

The DMD gene is made up of 79 exons, and mutations in that code can result in a dystrophin deficiency, which is responsible for the muscle wasting in DMD. Exon-skipping drugs are used to patch the ...

Participants in the FORWARD-53 trial had clinically significant improvements in time-to-rise, among other metrics.

PGN-EDO51 is designed to skip exon 51 of the dystrophin transcript, an established therapeutic target for approximately 13% of DMD patients, thereby aiming to restore the open reading frame and ...

Data on muscle delivery, exon skipping, dystrophin production and creatine kinase levels were assessed from seven participants in the 5 mg/kg cohort, 10 participants in the 10 mg/kg cohort ...

PGN-EDO51 acts by targeting exon 51 in the dystrophin gene. The therapy binds to RNA and efficiently interferes with gene expression in a sequence-specific manner. A functional form of the ...

Wave Life Sciences announced positive data from the Phase 2 FORWARD-53 trial of WVE-N531, which is an exon skipping oligonucleotide being ...

Del-zota is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) to skeletal muscle and heart tissue for the purpose of skipping exon 44 of dystrophin mRNA to enable the coding of ...