For Duchenne Moms Who Pushed for Cures, New Breakthrough Therapy Can’t Rebuild What’s Lost

June 30, 2023

(STAT News) – For Furlong and an entire generation of parents, the approval of Sarepta Therapeutics’ gene therapy, Elevidys, is an ecstatic moment, but one laced with pain. They mobilized for years to lay the groundwork, raising money for basic research, meeting with lawmakers, lobbying the FDA and educating biotech CEOs. Their sons participated in study after study, establishing foundational knowledge both Sarepta and regulators relied on. (Read More)