The company plans to submit a biologics licence application to the US Food and Drug Administration later this year.

The interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain ...

RGX-202, a DMD gene therapy, has been well tolerated in an ongoing clinical trial and improved motor function for DMD boys, ...

The Phase I/II Deliver trial sought to establish the impact of DYNE-251 on several mobility-related endpoints, seeing an ...

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...

Having found that in biomedical research, the Bird Rock resident and scientist at La Jolla’s Sanford Burnham Prebys has ...

After some earlier data sparked questions from analysts, Avidity Biosciences has released more data for its RNA-based ...

Avidity Biosciences (RNA) announced del-zota topline data from the Phase 1/2 EXPLORE44 trial in people living with Duchenne muscular dystrophy ...

Precision BioSciences (DTIL) announced the presentation of preclinical data for its PBGENE-DMD development program for the treatment of ...

The preclinical data highlights the ability of ultrasound-mediated delivery (UMD) to express nonviral genetic constructs in a redosable, durable manner, allowing full-length dystrophin expression ...

Neutrophil to Lymphocyte ratio (NLR) is a critical biomarker of health of astronauts during space mission, and that of aging ...