Great Britain was the first in the world to approve a revolutionary method of treating blood diseases
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The UK has authorized gene therapy for sickle cell disease and β-thalassemia in patients aged 12 and over. The state was the first in the world to officially approve this type of treatment.
This was reported by the Medicines and Healthcare Products Regulatory Agency of Great Britain (MHRA).
Sickle cell anemia and β-thalassemia are genetic diseases caused by errors in the genes for hemoglobin, which is used by red blood cells to carry oxygen around the body.
“Both sickle cell disease and β-thalassemia are severe lifelong diseases that can even be fatal in some cases.” MHRA Acting Director Julian Beach said in a statement.
| Photo: Spectral/Depositphotos |
The British regulatory body has approved the revolutionary treatment of these diseases with the drug Casgevy, which works by “editing” the gene that causes these ailments, writes Reuters.
Casgevy became the world’s first licensed drug that uses a gene editing tool. In 2020, it brought its inventors the Nobel Prize.
In clinical trials, Casgevy was found to restore healthy hemoglobin production in most participants with sickle cell anemia and transfusion-dependent β-thalassemia, alleviating symptoms of the disease, Beach added.
The MHRA said that the trials did not reveal any serious safety problems with the medicines.
Specialists who study genetic diseases hope that the use of Casgevy will eliminate the need for bone marrow transplants for people with these diseases. Previously, such transplants were the only possible treatment, even though the body could reject the donor bone marrow. Patients also need regular blood transfusions to treat anemia.
According to the Guardian, there are about 15,000 patients with sickle cell anemia in Great Britain. Another nearly 1,000 patients have β-thalassemia.
We will remind you that earlier we told that more than 50 bone marrow transplants from non-family donors were performed in Ukraine and how to become one of them.
Read also: What is it like to treat rare diseases in Ukraine
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