accelerated approval

DNA gene therapy gene editing helix CRISPR

Regenxbio starts pivotal study for DMD gene therapy

Regenxbio’s gene therapy improved muscle function for patients with DMD in a phase 1/2 study, with the biotech rolling the trial into a pivotal study.

Gabrielle Masson Nov 18, 2024 2:24pm

Wave surfs DMD success to regulators' doors, sending stock up

Sep 24, 2024 9:51am

Ultragenyx gets green light to seek FDA approval of gene therapy

Jun 13, 2024 5:55am

How Peter Marks plans to prevent '3 billion visits' to FDA

May 8, 2024 11:05am

FDA's Richard Pazdur explains confirmatory trial rejection, more

Apr 8, 2024 6:00am

Sarepta's next DMD drug seems to get the job done in phase 2

Jan 29, 2024 10:49am