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.

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

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

Hemophilia is a genetic disorder that limits the blood's ability to clot and affects around 30,000 people in the United States, mostly males. Left untreated, it can cause spontaneous bleeding ...

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 ...

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.

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, ...

A new gene therapy approved earlier this year can serve as a sustainable single-dose treatment for people with hemophilia B, ...

Beqvez (fidanacogene elaparvovec) led to about three times fewer bleeding episodes than preventive treatment with regular ...

Mullangi elaborated on the specific cancers linked to each of these gene mutations, emphasizing their varying degrees of risk and the broad spectrum of malignancies they influence, including rare ...

Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of ...