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Patients with beta thalassemia minor are usually asymptomatic and are often monitored without treatment. [8] Beta thalassemia minor may coexist with other conditions such as chronic hepatitis B, chronic hepatitis C, non-alcoholic fatty liver disease and alcoholic liver disease that, when combined or co-existing, may cause a person to have iron ...
Less serious side effects include gastrointestinal symptoms, which were found in 33% of patients in the first year of administration, but fell to 3% in following years; arthralgia; and zinc deficiency, with the latter being a problem especially for individuals with diabetes. [16]
There are two approved forms of gene therapy for beta thalassemia. [96] [97] Betibeglogene autotemcel, sold under the brand name Zynteglo, is a gene therapy for the treatment for beta thalassemia which adds a healthy beta-globin gene to the HSCs. [98] It was approved for medical use in the United States in August 2022.
Luspatercept, sold under the brand name Reblozyl, is a medication used for the treatment of anemia in beta thalassemia and myelodysplastic syndromes. [5] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [8]
Therefore, low compliance of patients is one of the major concerns of this therapy. Side effects include abnormal growth of bones and kidney damages. [9] [13] Deferoxamine is seldom used alone nowadays, but rather in combination with oral deferiprone to increase the effectiveness in reducing iron overload. [9] [13]
A separate study, published in the Journal of the American Academy of Dermatology looked at the effects of topical minoxidil in men with pattern hair loss over a total treatment period of 48 weeks.
Betibeglogene autotemcel, sold under the brand name Zynteglo, is a gene therapy for the treatment for beta thalassemia. [1] [5] [2] It was developed by Bluebird Bio and was given breakthrough therapy designation by the US Food and Drug Administration in February 2015.
Now, chatter about the side effects—nausea, ... This is the only FDA-approved topical treatment for hair loss and is available for women in 2 percent and 5 percent concentrations: “Minoxidil ...
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