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 ...

Jonathan D. Grinstein, PhD, Manfred Scholz, PhD, and Erik Wiklund, PhD, go behind the headlines to address a shifting policy landscape and what it means for the future of chronic disease, diagnostics, ...

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 ...

India has over 180 children with Hunter Syndrome, but many people are not even aware of this genetic disorder which mostly ...

CGT Global, a leading biotechnology company driving the development and delivery of innovative cell and gene therapies, today ...

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 ...