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.

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 ...

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 ...