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 ...

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 ...

When Sydney couple Adriana and Adam Sharpe welcomed their first child, they had no way of knowing she would go on to make history, not once, but twice, by the time she was four weeks old.After a ...

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

Novo Nordisk will advance its acquired ATTR amyloidosis with cardiomyopathy monoclonal antibody (mAb) to Phase III trials.

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

Ann and her husband, Simon, fear for their son’s life as the condition can be fatal if untreated. They are appealing for ...

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

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