Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death ...

Indicated for patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, the drug could be used in around 13% of the disease population. Sarepta’s share price almost ...

The drug was cleared to treat DMD patients amenable to exon 51 skipping – a group which represents about 13% of the population with the rare muscle-wasting disease. “For eteplirsen ...

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 ...

The DELIVER trial is evaluating DYNE-251 in individuals with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping, and updated results from the trial are being presented this ...

Wave Life Sciences Ltd. shares promising updates on AATD & DMD treatments. Click here for my updated look at WVE stock ...

A functional form of the dystrophin protein is produced by skipping exon 51, which should improve the disease condition. If approved, GlobalData projects the drug’s sales will reach $136m by 2030.

in patients with confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping; VYONDYS 53 for the treatment of Duchenne in patients with confirmed mutation of the dystrophin gene ...

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

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 ...