Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...

One thing that sticks with me is how earnest the documentary feels. Steen was both a dear and considerate friend to the ...

"I've read messages from men that say ‘I haven't cried since '99, and this is the first time I cried since then.'" ...

Since the isolation of the gene that causes Duchenne muscular dystrophy (DMD), scientists have progressed in understanding the mechanisms that lead to muscular diseases that can be evident from the ...

Regenxbio has revealed plans to submit a biologics licence application (BLA) for RGX-202, its investigational gene therapy ...

Novartis had one eye on the struggles its peers have encountered in the muscular dystrophy space when it decided to acquire ...

The agency's openness to a targeted pivotal study shows it’s still willing to consider accelerated clearance for Duchenne ...

Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for ...

Ernie and Cheryl Johnson had planned to hold onto their late son Michael's quirky key collection forever. But then they met 6 ...