Thelansis

Pediatric congenital athymia is an ultra-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 off infections. Multiple genetic abnormalities, congenital syndromes, and environmental factors are being associated, and the care of these infants is complex. While 22q11.2 deletion-associated with DiGeorge Syndrome (DGS) is the most common genetic defect associated with congenital athymia. FOXN1, PAX1, and others have also been identified as potentially causative. Children with congenital athymia may have repeated, often life-threatening infections because they don’t have enough working T cells (infection-fighting white blood cells) to fight them off. It’s important to know that these infections can be fatal.   The competitive landscape of Pediatric Congenital Athymia includes country-specific approved and pipeline therapies. Any asset/product-specific designation or review and Accelerated Approval are track...