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

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

Regenxbio’s investigational gene therapy improved muscle function for patients with Duchenne muscular dystrophy (DMD) in a phase 1/2 study. | Regenxbio’s investigational gene therapy improved muscle ...

A small trial of an experimental vaccine has shown promising results for patients with the most aggressive type of breast ...

In mice with muscular dystrophy, the treatment restored large therapeutic muscle proteins to normal levels, the researchers ...

Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic ...

PTC Therapeutics has received FDA approval for a gene therapy to treat AADC deficiency, a rare genetic disorder that impairs ...

Asian patients tend to present with heart failure at a younger ... which accounts for the remaining half of all HF cases) and Duchenne muscular dystrophy-associated cardiomyopathy, respectively.

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Regenxbio reported improved muscle function in boys with Duchenne muscular dystrophy using their gene therapy RGX-202. The ...