Carefully controlled schedules. Undercurrents of uncertainty. Persistent pain. People who are living with hemophilia B, and those who love them, face a lifetime of managing the complexities of this ...

BBM-H901 was shown to significantly reduce bleeding rates among hemophilia B patients in clinical trials, a new study in China shows.

China recently approved its first gene therapy for the rare genetic disorder hemophilia B, drawing renewed attention to this often-overlooked condition. Hemophilia is a rare but serious inherited ...

(BPT) – It was a diagnosis no parent ever wants to hear, and few parents have heard it. Mandy and Ryan Aberegg’s world shifted on its axis when their son Cooper, now 13 years old, was diagnosed ...

BOSTON--(BUSINESS WIRE)--hC Bioscience, a biopharmaceutical company developing a fundamentally novel approach to treating genetic diseases through tRNA-based protein editing, today announced ...

Pfizer’s first FDA-approved gene therapy is the second such treatment for the inherited bleeding disorder hemophilia B, introducing some competition to a therapeutic area that now has two of the most ...

Hemophilia A and B are similar conditions that affect the blood’s ability to clot. They typically occur due to gene variations that affect different blood clotting factors. Hemophilia is a group of ...

CRISPR/Cas9, a powerful genome editing tool, is showing promise for efficient correction of disease-causing mutations. For the first time, researchers from the Perelman School of Medicine at the ...

The FDA approved the first gene therapy treatment for adults with severe hemophilia A. Roctavian is an adeno-associated virus vector-based gene therapy that is administered as a single dose by ...

If you know anything about hemophilia, it’s probably that the condition was rampant in the British royal family in the 19th and early 20th centuries. Queen Victoria, who had nine kids, was highly ...

MCDONOUGH, Ga. - Stormy Johnson, of McDonough, says she knew something was very wrong back in 2002, when she woke up one morning and found blood in her bed, where her then 3-year-old son Collin had ...