Abu Dhabi set to begin clinical trials of Mitapivat to cure thalassaemia


(MENAFN) Abu Dhabi is set to begin clinical trials of Mitapivat, a drug that could have significant implications for the treatment of thalassaemia in the UAE.

Thalassaemia is a group of inherited blood disorders that results in the body producing insufficient levels of haemoglobin, a protein that carries oxygen in the blood. This can cause patients to become anaemic and experience symptoms such as fatigue, shortness of breath, weakness, slow growth, changes to the bones of the face, and changes in skin colour and urine. People with thalassaemia may also be more susceptible to infections.

There are two main types of thalassaemia, alpha and beta, depending on which part of the haemoglobin molecule the body is less able to produce. The disorder is particularly prevalent in regions such as the Middle East, Asia, and the Mediterranean, with an estimated 7% of the world’s population carrying a thalassaemia gene.

The drug Mitapivat is currently in phase 3 clinical trials, with Burjeel Medical City in Abu Dhabi participating in the study. The trials will assess the drug’s safety and effectiveness in treating adults with thalassaemia. If successful, Mitapivat could offer a significant step forward in the treatment of the disorder. It works by targeting a specific enzyme that regulates the production of haemoglobin, helping to increase the levels of this protein in the blood.

The clinical trials in Abu Dhabi are just one example of the ongoing efforts to find effective treatments for thalassaemia. While there is currently no cure for the disorder, treatments such as blood transfusions and bone marrow transplants can help manage the symptoms. However, these treatments are not without risks, and there is a need for more targeted and less invasive therapies.

Mitapivat offers a potential solution, with the drug showing promising results in previous trials. In a phase 2 study, the drug led to significant increases in haemoglobin levels among patients with thalassaemia. If the phase 3 trials are successful, Mitapivat could provide a safer and more effective treatment option for patients in the UAE and beyond who are living with this debilitating disorder.

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