(Mirror Daily, United States) –A cure for hemophilia is still far off, but today, the Dutch announced they have a hemophilia working treatment developed by UniQure, also a Dutch company. Trials are still made for this miracle therapy for hemophilia B, but hopes are high as patients around the world hope for an improvement.
Hemophilia B is a disorder that affects blood clotting. It is caused by a mutation in the gene that produces factor IX, a serine protease that is involved in the process of blood coagulation. It is a widely spread disorder that affects an important number of people. The main concern in patients that suffer from it is bleeding to death because of a small injury due to the fact that the blood is not able to clot as it would normally.
The AMT-060 is an experimental gene therapy that was applied to a sample of 20 patients. The treatment is still experimental and it has a lot more ground to cover before being declared efficient and safe to use. In order for that to happen, patients that received this type of gene therapy must display no more signs of affection, and must overcome the need for their current hemophilia medication.
The reason for the early celebration is the state in which patients that received the treatment 12 to 20 weeks ago present themselves. Their body managed to produce a significant amount of factor IX (somewhere around triple the quantity they were producing prior to the treatment).
This is very important, and extremely good news for the patients because any risen amount of protein IX in the blood of a hemophiliac is a reason for celebration.An increase in the levels of protein IX in the blood of a person that suffers from hemophilia B can tell the difference between life and death for a patient.
AMT-060 is such good news because gene therapy is a much simpler way of handling the disease as opposed to the present treatment that consists in regular injections with the coagulatory factor IX.
Of course, research is yet to be made because the sample of patients treated was so small. People are different in various ways and that means that though some have responded in a positive manner to the treatment, others may not. And these are important factors to be taken in consideration. Also, they must be careful to avoid violent responses of the patient’s immune system when inserting the therapy gene.
UniQure is not the only company that is working with gene therapy at the moment, but it is the only one experimenting with a virus that is adeno-associated. More information on the research conducted by UniQure will be available in the second quarter of this year.
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