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

LEXINGTON, Mass. and AMSTERDAM, Oct. 15, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing ...

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, ...

This suggests that people with ALS have a greater chance of carrying gene mutations associated with ... once-weekly drug treatment regimen for hemophilia Oct. 11 (UPI) -- Patients suffering ...

But when she ended up with just two viable embryos—one with the mutation ... genetic diseases linked to the X chromosome, Duchenne is among the more common, along with certain forms of hemophilia.

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 ...

25, 2024 — Adults with hemophilia ... mutations in microRNA, by ... Researchers Discover Potential Mole Reversal Therapy in Rare Condition June 17, 2024 — Researchers have designed a new ...

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

Von Willebrand Disease in dogs is a complex clotting disorder that can vary in symptoms and the risk of genetic inheritance.

Discover the top biotech companies pioneering the next healthcare breakthroughs. Learn how innovative firms are ...

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 ...