Congenital athymia
Congenital athymia is a rare immune disorder in which a child is born without a thymus – an organ that plays a critical role in helping the body learn to fight infections.[1] Children impacted by this disease typically die within the first two years of life and may have repeated, often life-threatening infections because they lack adequate working T cells (a kind of infection-fighting white blood cell).[1]
Treatment
In October 2021, the thymus tissue product Rethymic was approved by U.S. Food and Drug Administration (FDA) as a medical therapy for the treatment of children with congenital athymia.[1] It takes six months or longer to reconstitute the immune function in treated children.[1]
References
- 1 2 3 4 "FDA Approves Innovative Treatment for Pediatric Patients with Congenital Athymia". U.S. Food and Drug Administration (FDA) (Press release). 8 October 2021. Retrieved 8 October 2021. This article incorporates text from this source, which is in the public domain.
This article incorporates public domain material from the United States Department of Health and Human Services website http://www.fda.gov.
Further reading
- Hsieh EW, Kim-Chang JJ, Kulke S, Silber A, O'Hara M, Collins C (August 2021). "Defining the Clinical, Emotional, Social, and Financial Burden of Congenital Athymia". Adv Ther. 38 (8): 4271–4288. doi:10.1007/s12325-021-01820-9. PMC 8342356. PMID 34213759.