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

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

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

The firm will seek accelerated approval for WVE-N531 in DMD amenable to exon 53 skipping based on FDA feedback and Phase II data.

Wave Life plans a 2026 NDA filing for WVE-N531 after positive Phase 2 data in Duchenne muscular dystrophy, showing functional gains and biomarker improvements.

Avidity Biosciences, Inc.'s innovative AOC platform and promising clinical programs make it a high-risk, high-reward ...

As it gears up to submit an approval application next year, Wave Life Sciences has presented fresh phase 2 data showing its ...

An experimental gene therapy for Duchenne Muscular Dystrophy (DMD) has showed better-than-expected results in a three-patient trial, according to preliminary data. Company shares jumped 60 percent ...

RGX-202, aims to address the root cause of DMD by delivering a functional copy of the gene that encodes microdystrophin, a protein that is missing or defective in DMD patients. DMD is a form of ...

The death this week of a teenager receiving Sarepta Therapeutics’ gene therapy Elevidys for Duchenne muscular dystrophy is a tragic reminder of the stakes involved in cutting-edge biotech ...

Credit: Shutterstock / ChiccoDodiFC. Regenxbio has reported positive interim data from the Phase I/II portion of its Affinity Duchenne study (NCT05693142), which is examining its investigational gene ...

RGX-202, a one-time gene therapy designed to treat Duchenne muscular dystrophy (DMD), has been well tolerated in an ongoing clinical trial, with no serious side effects reported. Interim data from the ...