Dyne is eyeing an accelerated approval filing for DYNE-251 in early 2026 that would pit the asset against Sarepta’s Exondys ...

Avidity Biosciences (RNA) announced del-zota topline data from the Phase 1/2 EXPLORE44 trial in people living with Duchenne muscular dystrophy ...

Mizuho views the selloff in shares of Sarepta (SRPT) yesterday due to concerns about Elevidys adoption as a result of the fatal acute liver ...

Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics ...

A University of Alberta research team has taken the first step toward proving its innovative gene therapy can successfully ...

PepGen has previously faced an FDA clinical hold on the investigational new drug application of PGN-ENO51 in the Phase II ...

GenAssist’s IND application for GEN6050X receives US FDA clearance, a globally first-in-class base editing drug for duchenne muscular dystrophy: Suzhou, China Friday, March 7, 2 ...

Taiho is parting with $400 million upfront for Araris, a spin-off from the Paul-Scherrer-Institute in Zurich, Switzerland, ...

Nearly half of those living with Duchenne who are amenable to exon 44 skipping are adults. ELEVATE-44-102 will provide clinical experience from this important population for our growing data package ...

Dyne Therapeutics’ DYNE-101 shows positive results in phase 1/2 ACHIEVE study. Read why DYN stock may rise with FDA approval and a $2.78B market opportunity.

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 ...

Edgewise Therapeutics' stock soars on positive Becker Muscular Dystrophy trial results. Learn why EWTX is a promising buy with derisked programs.