This week saw both Regenxbio and Solid Bio report new data with their gene therapy candidates for Duchenne muscular dystrophy ...

The investigational gene therapy SGT-003 is showing early signs of safety and efficacy for Duchenne muscular dystrophy in a ...

Solid Biosciences’ SGT-003 is the only late‑stage program to show early cardiac benefit across biomarkers and function, according to William Blair.

Solid Biosciences (SLDB) reported updated “positive” interim data from the ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial of SGT-003. The new data, including additional muscle biopsy, serum ...

The firm is hoping to outline a potential path to accelerated approval for SGT-003 as it gears up to launch a randomized, controlled Phase III trial.

In patients with baseline cardiomyopathy, Deramiocel demonstrated an even greater treatment effect on cardiac function. In this subgroup, treatment resulted in a 3.3 percentage-point improvement in ...

Investigational RGX-202 continues to demonstrate evidence of positively changing disease trajectory for Duchenne Pivotal dose participants exceeded external controls across functional ...

March 11 (Reuters) - Regenxbio said on Wednesday that interim data from a early-to-mid stage study of its experimental gene therapy in patients with Duchenne muscular dystrophy showed continued ...

SGT-003 in the INSPIRE DUCHENNE trial to date - - Robust microdystrophin expression with consistent mechanistic evidence of dystrophin associated protein complex (DAPC) restoration and improvements in ...

People with BMD who received sevasemten in clinical trials maintained stable motor function over several years of follow-up, new data show.

Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...

The MESA data show stabilization of function in participants on sevasemten versus the predicted functional decline observed ...