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 ...

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 ...