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 ...

The procedure is a major step toward what Daniel Cressy hopes is a long-lasting cure for the rare genetic blood disorder.

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

GAAP revenue of $14.2 million for Q2 2025 beat analyst estimates and Revenue more than doubled year over year in the second quarter, beating analyst estimates. Net loss per share (GAAP) narrowed to ...

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

Q2 2025 Earnings Call Transcript August 6, 2025 Charles River Laboratories International, Inc. misses on earnings ...

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

Changes in how brain cells generate energy may drive the development of Alzheimer's disease and influence how patients ...

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

A 26-year-old Downriver man seen at Children’s Hospital of Michigan in Detroit since infancy for a rare bleeding disorder in ...