They find molecules to improve hemophilia (+ details)

(Caracas, July 07. News24) .- Treatments with a special molecule (Emicizumab) whose use is approved in phases in Latin America are increasing options to improve the quality of life of hemophiliac patients several experts agreed at a forum held this week in Cali (Colombia).

"New studies have been done that allow us to use new molecules, we have molecules like monoclonal antibodies (a type of protein produced in the laboratory that can bind to a substance in the body) and There is also a genetic therapy where very interesting advances are made for hemophilia A and B. "Efe Miguel Antonio Escobar, Medical Director of the University of Texas Research Center

Escobar participated in this week at the seventh edition of Roche Press Day, an information meeting and education that promotes the continuing education of journalists and where the latest developments in the health sector in Latin America are reported. [19659004] There are about 500,000 people in the world suffering from hemophilia, a disease that consists of the absence of a blood protein that allows clotting. & Amp; haemophilia A, which accounts for 85% of cases, there is a lack of what is called factor VIII and for B (one who suffers from the remaining 15%), there is low or nonexistent percentages of factor IX.

For blood clotting to be possible, a total of 13 factors must be involved together.

"You have to think about the coagulation as if it was a symphony. If a part is missing, it does not work well," says Escobar, who works as a hematologist at the Center for Research. Hemophilia and thrombophilia of the Gulf States of the University of Texas (United States).

"One of the molecules (Emicizumab) has recently been approved, clinical studies have shown that it is very safe and effective from a medical point of view.It prevents hemorrhages, which is our goal in haemophiliac patients, "says Escobar

Daniel Ciriano, medical director of Roche, the pharmaceutical company that developed the molecule, told Efe that it would be administered to patients who develop Inhibitors of Coagulation Factor VIII

"Today, we are replacing factor VIII with a factor VIII that the body does not produce. Continuous administration means that 30% of patients develop antibodies that block it, inactivate it (with factor VIII), "he added.

The new treatment made patients work with inhibitors developed and the annual bleeding rate is controlled

"In patients treated with the molecule, the annual bleeding rate is almost nil", explains Ciriano.

Quality of life and patient adherence the treatment also improves, since people, including children should not inject two or three times a week to give themselves the factor they need.

"When the injections are done once a week, and nowadays studies show that they can be done every two or four weeks, these are benefits enjoyed by patients."

Escobar also pointed out that s are already in the phase of clinical studies some of the Lamadas "gene therapies " which consist of "choosing a vector, the badociated adeno 8, to which genetic modifications are made".

"Then it is injected into the individual suffering from hemophilia and the liver manages to produce factor VIII or IX," says the specialist

Hemophilia is a disease genetics which affects the male population (99% of cases ) and which is transmitted from mother to child

.Latinina is estimated that there are about 58 000 cases, of which 52% have not yet been diagnosed.Creducing this number is the goal of the World Federation of Hemophilia (WFH).

For fighting the under-diagnosis, the federation is working with the countries of the region where they found disparities in terms of data.] "Guatemala, Bolivia and Paraguay have the largest number of subregisters. We have good results in Brazil, Argentina, Venezuela, Costa Rica, Chile and Panama, "said César Garrido, board member of the WFH, an international non-profit organization founded in 1963 by a worldwide network of patient organizations. in 140 countries

Until 1937, the life expectancy of a person with hemophilia was 13 years . Today, the average fluctuates between 65 and 70 years old.

With information from EFE

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