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 ...

PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies ...

PepGen is 'temporarily pausing' a Phase 2 study of its exon 51 skipping drug for Duchenne muscular dystrophy, it said Tuesday ...

Oral Presentations Title: CONNECT1-EDO51: A 12-Week Open-Label Phase 2 Study to Evaluate PGN-EDO51 Safety and Efficacy in People with Duchenne Amenable to Exon 51 Skipping Session: Clinical Trial ...

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 ...

CONNECT1 has enrolled two cohorts of boys and young men living with DMD amenable to exon 51 skipping and its endpoints include safety and tolerability, dystrophin production, exon skipping ...

It offers EXONDYS 51 injection to treat duchenne muscular dystrophy (duchenne) in patients with confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping; VYONDYS 53 for the ...

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 ...