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World Hemophilia Day: Gene Therapy for Hemophilia

World Hemophilia Day, which is observed on the 17th of April (every year) is recognized to increase awareness about hemophilia. Hemophilia is a rare disorder in which one’s blood doesn’t clot due to insufficient blood clotting nutrients, it is usually inherited. So if a person is suffering from hemophilia, may bleed for a longer time (than usual) after an injury or surgery.

Haemophilia is caused due to the change or mutation of a gene, that gives instructions to the clotting factor proteins to form a blood clot. Usually being in the F8 or F9 genes which causes either hemophilia A(HA) or hemophilia B(HB). People with HA have a shortage of the clotting factor VII and HB patients lack IX clotting factor. Patients with a serious condition of this disease are vulnerable and easily inclined to internal bleeding, which mainly occurs in the elbows, knees, and ankles ( spontaneous bleeding). This bleeding can lead to tissue and organ damage that can be deadly if not treated. ​Crucial efforts are taken to increase awareness for better diagnosis and access to care for the millions who remain without treatment through World Hemophilia Day.

Signs and symptoms of severe disease include, according to the Mayo Clinic:

  • Unexplained and excessive bleeding from cuts or injuries, or after surgery or dental work
  • Deep bruises
  • Unusual bleeding after vaccinations
  • Pain, swelling, or tightness in your joints
  • Blood in your urine or stool
  • Nosebleeds without a known cause

Brain hemorrhage
A slight bump to the head can cause hemorrhage within the brain for many suffering from severe hemophilia. Occurrences of this specific trauma are rare. Symptoms:

  • Painful, prolonged headache
  • Repeated vomiting
  • Lethargy
  • Double vision
  • Sudden weakness
  • Convulsions or seizures

There is no cure for hemophilia. Each of the new gene therapy treatments is administered just once a single injection or infusion that can dramatically improve a patient’s life. Although more study is needed, early results regarding the durability of response to these treatments is positive. The health experts believe that nearly 80 percent of Indians with serious blood disorders are not diagnosed due to the absence of proper diagnostic facilities in remote areas. India is estimated to have the second-highest number of patients with hemophilia. Every year, events are conducted throughout the world to mark the significance and to spread awareness about this disease. But, this year, the pandemic has made social distancing, a thriving need. So, World Haemophilia Day will be observed by getting involved virtually and celebrating the hard work of medical professionals in providing treatment for all.

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