PepGen Inc. , a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological ...

Learn more about whether Ionis Pharmaceuticals, Inc. or PTC Therapeutics, Inc. is a better investment based on AAII's A+ Investor grades, which compare both companies' key financial metrics.

Top-line Phase 3 trial data show ambulatory boys with Duchenne continued with motor improvements two years after a single ...

Columnist Shalom Lim is working with Julia Cameron's popular book "The Artist's Way" as a route to develop his creative self, ...

Donald Trump continues to make waves in biopharma; Sage rejects Biogen’s unsolicited takeover offer; the obesity space sees ...

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...

The positive results from the second part of the EMBARK trial contrast with findings from one-year post treatment, which did ...

Tazeen Ahmad, an analyst from Bank of America Securities, maintained the Buy rating on Sarepta Therapeutics (SRPT – Research Report). The ...

(RTTNews) - Sarepta Therapeutics, Inc. (SRPT) Monday reported positive topline results from Part 2 of the Phase 3 study, dubbed EMBARK, of its gene therapy Elevidys approved for the treatment of ...

The data showed reduced difficulties in standing, walking and running that were statistically significant, the company said.

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Prophylactic cardiac treatment may prolong survival in Duchenne muscular dystrophy (DMD), but only one-quarter of individuals ...