Ad
related to: beta thalassemia treatment options in ohio map location image- For Patients
Visit this website if you are a
patient seeking information.
- Emerging NTD Data
See how hemoglobin levels
affect NTD patients.
- Complications
Discover and explore thalassemia
symptoms and complications.
- Management
Find the latest thalassemia
management guidelines.
- For Patients
Search results
Results from the WOW.Com Content Network
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.
The treatment was approved in the United Kingdom for the treatment of transfusion-dependent beta thalassemia in November 2023 [38] [39] [40] and in the United States in January 2024. [ 41 ] [ 42 ] [ 43 ]
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.
The groundbreaking treatment can now also be used to treat transfusion-dependent beta thalassemia in people 12 and older. Like sickle cell, beta thalassemia is an inherited blood disorder.
The $100-million, 55,000-square-foot center is the first treatment facility in central Ohio to offer this type of radiation therapy.
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 ...
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]
Alpha thalassemia patients have partial or complete defects in alpha globin production, leading to a relative abundance of beta globin chains in the cell. These excess beta globin chains aggregate to form HbH , which has decreased solubility and precipitates in the red blood cell cytoplasm . [ 10 ]
Ad
related to: beta thalassemia treatment options in ohio map location image