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.

Solid Biosciences Inc. shares dropped more than 8% on Wednesday after the company released data from its Duchenne gene therapy trial. Trading in the stock was briefly halted earlier in the day. The ...

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

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 ...