During the first half of 2025, we achieved target enrollment in our ongoing gene therapy clinical trials of TN-201 for MYBPC3-associated HCM and ...

After a second Elevidys patient death from acute liver failure, Sarepta in July launched a major restructuring that involves ...

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

Sarepta Therapeutics (SRPT) stock in focus as report says company hired a Trump-linked lobbying firm after deaths linked to ...

In this week’s edition of InnovationRx, we look at a biotech entrepreneur treating a rare genetic disease, new tech aimed at ...

Uppsala University Hospital-led investigators report that gene-edited donor islet cells survived 12 weeks inside a man with ...

Connecticut is now participating in a new federal program aimed at expanding treatment access for patients with sickle cell ...

A combo of cancer drugs letrozole and irinotecan has been identified — via a complex AI-driven study — to reverse memory ...

Key Points Non-GAAP earnings per share reached $0.74, surpassing estimates by $0.03 (non-GAAP), but GAAP revenue of $181.1 million missed expectations and rose 1.7% (GAAP) compared to the second ...

At only 6 months, Gerard and Kaitlin Norton’s daughter Madeline was diagnosed with PKAN, a rare neurodegenerative disorder ...

Ocugen says it is on track to reshape the market for gene therapies against eye disorders over the next three years, by ...

From alveolar injury to extracellular matrix accumulation, the complex biology of pulmonary fibrosis is being unraveled ...