| Title | Gene Therapy for Beta-Thalassemia Blood Disorder | |
| Date | Thursday April 19 2018, @03:04PM | |
| Author | Fnord666 | |
| Topic | ||
| from the positive-results dept. | ||
Gene Therapy For Inherited Blood Disorder Reduced Transfusions
Gene therapy is showing promise for treating one of the most common genetic disorders. Results of a study published Wednesday show that 15 of 22 patients with beta-thalassemia who got gene therapy were able to stop or sharply reduce the regular blood transfusions they had needed to alleviate their life-threatening anemia. There were no serious side effects.
[...] The researchers stress, however, that more research is needed to fully evaluate how well the treatment works and how safe it is. Still, the company that's developing the treatment, Bluebird Bio of Cambridge, Mass., plans to seek approval of the treatment in Europe by the end of the year, a spokeswoman said in an email.
An estimated 288,000 people have beta-thalassemia worldwide, which makes the disease one of the most common genetic disorders. It's found most often in Mediterranean countries, the Middle East, Asia, India, and parts of Africa and South America. In the United States, about 10,000 to 15,000 patients have beta-thalassemia.
Also at BBC.
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia (DOI: 10.1056/NEJMoa1705342) (DX)
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printed from SoylentNews, Gene Therapy for Beta-Thalassemia Blood Disorder on 2024-04-20 03:46:43