Spanish authorities issued a positive recommendation for national reimbursement of Hemgenix gene therapy for eligible ...

LEXINGTON, Mass. and AMSTERDAM, Oct. 15, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, ...

The approval of Hympavzi makes it the first treatment for both hemophilia A and B to be administered through a pre-filled pen ...

The approval of Hympavzi Friday was a dose of good news for Pfizer, which is dealing with research setbacks and a challenge from an activist investor.

Columnist Alliah Czarielle clears up some common misconceptions about what happens when people with hemophilia have minor cuts or abrasions.

Pfizer's Hympavzi receives FDA approval as the first anti-TFPI therapy for hemophilia A and B. This subcutaneous, once-weekly ...

A new study has punctured a longstanding assumption about the source of the most common type of DNA mutation seen in the genome--one that contributes to many genetic diseases, including cancer.

Researchers from the Yong Loo Lin School of Medicine, National University of Singapore (NUS Medicine) are working on a ...

Wendy Fry had just celebrated her first Christmas with her first child, 8-month-old Penny, when she woke the next day to another first: Her daughter having her first seizure. "I have never felt ...

She decided to get pregnant using IVF so that she could select embryos without the mutation for ... Of the hundreds of genetic diseases linked to the X chromosome, Duchenne is among the more common, ...

These work by replacing or deactivating disease-causing mutations. This is a newer treatment area with potential risks, but it may eventually provide radically improved management for various genetic ...

Patients had fewer bleeds after treatment with Beqvez compared to standard treatment, but questions remain about which ...