Neuroblastoma can be a particularly insidious cancer. In about half of all cases, tumors regress, even without therapy. In ...

After telegraphing an investigation in November, the FDA has moved to restrict the use of bluebird bio’s gene therapy Skysona ...

In this webinar, Martin Jarrold, Benjamin Clarke, and Rebecca D’Esposito will discuss the impact of charge detection mass ...

After a strong launch for its cancer gene therapy, Ferring Pharmaceuticals is setting the stage for long-term growth.

Net loss per share (GAAP) was $0.62, but remained negative as the company continues to invest heavily in research and development. The pivotal Phase 2 trial for Danon disease was placed on clinical ...

Sarepta Therapeutics Inc.’s shares soared after the US biotech reported revenue that beat estimates, which analysts ...

Are we doomed to repeat the mistakes of our parents? “Therapist Zero” is the story of a father determined to avoid the ...

This is the web edition of D.C. Diagnosis, STAT's twice-weekly newsletter about the politics and policy of health and ...

Sarepta did not hold an investor call for its second-quarter earnings report or provide an updated full-year revenue outlook.

The FDA's approval of the first CRISPR-Cas9–based gene therapy marked a major milestone in biomedicine, validating genome ...

After decades of strong federal support for scientific research, the US government is now proposing deep cuts. Here’s what ...