Transforming Hemophilia Management: Gene Therapy's Potential
Hemophilia , a rare but severe bleeding disorder, has long been a challenge for patients and healthcare providers alike. It is primarily characterized by impaired blood clotting due to a deficiency in clotting factor proteins. Hemophilia can be classified into several types, including Hemophilia A, Hemophilia B, Hemophilia C, and even Von Willebrand Disease. For years, patients have relied on frequent infusions of clotting factor concentrates to manage their condition. However, recent breakthroughs in gene therapy, such as Fidanacogene Elaparvovec, have opened up new possibilities for hemophilia treatment. Understanding Hemophilia Hemophilia is an inherited disorder, typically affecting males. Hemophilia A and Hemophilia B are the most common forms, caused by deficiencies in clotting factors VIII and IX, respectively. Hemophilia C, though rarer, involves a factor XI deficiency. Von Willebrand Disease, on the other hand, is characterized by a deficiency or dysfunction of von Willebrand...