- 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.

(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 ...

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At a mean age of 9 years old, ELEVIDYS-treated patients achieved mean North Star Ambulatory Assessment (NSAA) scores above baseline three years after treatment (n=52) ELEVIDYS gene therapy ...

Pfizer Inc. (NYSE:PFE) is one of the Most Undervalued Long Term Stocks to Buy Right Now. On October 19, Pfizer Inc. (NYSE:PFE) and Astellas Pharma US Inc. announced final overall survival results from ...

Pfizer Inc. (NYSE:PFE) and Astellas Pharma Inc. (OTC:ALPMF) (OTC:ALPMY) on Thursday released topline results from the overall survival (OS) analysis from the Phase 3 EMBARK study evaluating Xtandi ...