Congenital athymia is a rare pediatric disease characterized by the absence of functional thymus. Resulting from a defect in FOXN1 and PAX 1 genes , these patients have profound immunodeficiency, high susceptibility to infection, and, frequently, autologous graft-versus-host diseases. Cultured thymus tissue was being investigated as a potential treatment option for this condition. Recently, the USFDA has approved Rethymic for the treatment of pediatric patients with this condition.
Key features Children born with congenital athymia typically die within the first two years of life and may have life-threatening infections during their limited life span. Rethymic is composed of human allogeneic thymus tissue, which is processed, cultured, and then implanted into patients to help reconstitute immunity. The dose is customized according to every patient, the surface area of the Rethymic…
