Sebastien Beauzile, 21, is the first New Yorker to have received the breakthrough Lyfgenia treatment, according to the New ...

Shares of Sarepta Therapeutics SRPT plunged nearly 24% last week after the company reported the death of a patient following ...

The Company is currently analyzing the case and will update the prescribing information for Elevidys to note this development.

Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...

Why didn’t analysts share the same fear that gripped investors about Sarepta stock? One reason is the known risk of acute liver injury associated with adeno-associated virus vector (AAV)-based gene ...

Ractigen Therapeutics, a clinical-stage biopharmaceutical company pioneering small activating RNA (saRNA) therapeutics, today announced the presentation of positive preliminary data from its ongoing ...

Skin cancer advanced clinical practitioners ensure continuity of care for patients with malignant melanoma and other advanced ...

First and only immunotherapy to show meaningful overall survival benefit in a Phase 2 trial in patients with advanced ovarian cancer Initiation of trial sites underway for ground-breaking Phase 3 pivo ...

It's early days for xenotransplantation, but eGenesis, Eledon, United Therapeutics and more are working to develop solutions ...

Scientists have described antimicrobial resistance as an overlooked pandemic. Improving surveillance can help prevent deadly ...

Scientists from Johns Hopkins Medicine and eight other institutions in the United States, Africa and Europe say they have ...