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

Avidity Biosciences, Inc.’s RNA share price has dipped by 7.10%, which has investors questioning if this is right time to buy ...

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.

Catalyst Pharmaceuticals (CPRX) reported that its sub-licensee in Canada, Kye Pharmaceuticals, has announced that Health Canada has accepted ...

DMD was first described by French neurologist Guillaume Benjamin Amand Duchenne in the 1860s. However, until the 1980s, ...

Kye Pharmaceuticals announced it has submitted a New Drug Submission, or NDS, to Health Canada for the regulatory review and approval of ...

Dec. 16, 2024 — A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration. Researchers have discovered that an ...

Giulio Cossu, MD, University of Manchester, discusses barriers to gene therapy research and development.

Researchers in pharma and beyond have historically glommed onto a limited number of disease targets, limiting innovation. AI ...

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