A mom was overjoyed when she learned a drug could save her baby's life. Then she learned it cost $2 million.

A mom was overjoyed when she learned a gene therapy treatment could save her child's life, until she found out no one would ...

New gene therapy drugs can save lives, but many cost millions

A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...

Is Sarepta Therapeutics a Bargain After Recent Share Rebound and DCF Valuation Check?

Wondering if Sarepta Therapeutics is a beaten down biotech bargain or a value trap? This article will walk through the numbers in plain English so you can decide with confidence. Despite a brutal long ...
Clinical Trials Arena on MSN

Dyne to seek accelerated approval of DMD drug on Phase I/II data

Dyne Therapeutics intends to submit a biologics licence application to the FDA for accelerated approval in Q2 2026.

Sarepta a new outperform at Wedbush as selloff overdone

Wedbush upgrades Sarepta Therapeutics, citing overdone selloff and potential upside as FDA could re-approval Elevidys for non ...
Los Angeles Business Journal

Arrowhead Hits the Mark

This article has been revised and clarified. Pasadena-based biopharma company Arrowhead Pharmaceuticals Inc. had a ...
BioSpace

Dyne’s Exon Skipper Delivers ‘Best Ever’ Functional Outcomes in Early DMD Study

Dyne’s exon-skipping therapy zeleciment rostudirsen resulted in an approximately sevenfold increase in dystrophin levels at ...
STAT

Dyne Therapeutics plans to file for approval for next-generation Duchenne drug

Dyne Therapeutics plans to submit its next-gen Duchenne muscular dystrophy drug to the FDA after a successful trial.

Dyne Therapeutics Announces Positive Topline Results from Phase 1/2 DELIVER Trial of Z-Rostudirsen in Duchenne Muscular Dystrophy (DMD)

Registrational Expansion Cohort (REC) met primary endpoint, demonstrating statistically significant increase in dystrophin to 5.46% at 6 months ...