For hemophilia B patients, frequent bleeding can easily lead to damage to joint structure and function ... an inherited bleeding disorder caused by the deficiency of the clotting protein factor IX ...

Discover how an emerging technology called tRNA therapy could become the next big thing in genetic disease treatment.

IMARC added that the FDA approved 3 novel gene therapies for conditions such as spinal muscular atrophy and hemophilia ... which encodes the protein dystrophin. Dystrophin is important for ...

Proteins form the structural and functional backbone of the cell, and any perturbation in their synthesis can disrupt normal ...

CSL Behring Austria today announced that the first haemophilia B patient in Austria was treated with the gene therapy HEMGENIX® (etranacogene dezaparvovec) at the Comprehensive Care Center of the ...

Qfitlia (fitusiran), an siRNA drug to treat the frequency of bleeding episodes in adult and pediatric patients with hemophilia A or B, gets FDA approval.

Haemophilia A is caused by the lack of a specific protein needed for blood to clot. Without it, even small injuries can lead to prolonged or spontaneous bleeding, especially into joints like knees ...