CureDuchenne, led by CEO Debra Miller, is at the forefront of research for Duchenne muscular dystrophy, which is a rare and fatal neuromuscular disease.

Explore Solid Biosciences’ potential with promising SGT-003 trial results for DMD, strong financials, and market recovery ...

A few weeks after Sarepta Therapeutics confirmed a patient died after receiving its drug Elevidys, research published April 3 ...

A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular ...

A new article argues dystrophin immunogenicity is an under-examined issue in the treatment of Duchenne muscular dystrophy.

New Gene Therapy Approach Shows Promise for Duchenne Muscular Dystrophy July 24, 2024 — Researchers have made a significant breakthrough in developing a new gene therapy approach that restores ...

Discover how an emerging technology called tRNA therapy could become the next big thing in genetic disease treatment.

Oppenheimer analyst Andreas Argyrides lowered the firm’s price target on Sarepta (SRPT) to $184 from $215 and keeps an Outperform rating on the ...

One day after the European Medicines Agency requested that three clinical trials of Elevidys be placed on hold after the ...

Sarepta (SRPT) and Roche (RHHBY) expect to submit data to address a clinical hold imposed on Elevidys, a gene therapy for ...

Therapeutics shared the following update related to ELEVIDYS, the only approved gene therapy in patients with Duchenne muscular ...

Sarepta Therapeutics said on Friday it had temporarily halted three trials testing its gene therapy Elevidys, following the death of a patient last month upon receiving the treatment.