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

Jaipur: SMS Hospital has established a department for treatment of blood-related cancers and other blood disorders. The ...

Enrollment Complete in Cohorts 1 and 2 of MyPEAK™-1 Phase 1b/2 Trial of TN-201 for MYBPC3-associated HCM; Positive DSMB ...

There's a fresh push to edit the genes of human embryos to prevent diseases and enhance characteristics that parents value.

Gene Mack, CEO, Gain Therapeutics, explains how the company’s Magellan AI platform analyzes novel protein binding sites to identify and design drug candidates beyond what’s currently available in the ...

Cash runway extended into 2028; expected to enable multiple meaningful clinical data read-outs -- APOE program added to ...

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 ...