- Study did not meet primary or secondary endpoints. - LIVMARLI was generally well-tolerated, with no new safety findings. FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: ...

Over a 52-week treatment period, the gene therapy demonstrated robust evidence for a clinically meaningful benefit, including across several prespecified functional secondary end points vs placebo.

Delandistrogene moxeparvovec-rokl showed significant functional benefits in ambulatory DMD patients compared to external controls in the EMBARK trial. The therapy is the only approved gene therapy for ...

(RTTNews) - Sarepta Therapeutics Inc. (SRPT), on Monday, announced positive topline three-year results from its EMBARK study, showing that ELEVIDYS significantly slowed disease progression across key ...

Good morning, and welcome to Sarepta's EMBARK 3-year data top line results call. As a reminder, today's program is being recorded. At this time, I'll turn the call over to Doug Ingram, Sarepta's Chief ...