Posted by Michael Wonder on 16 Nov 2024
AlveoGene receives rare paediatric disease designation from FDA for AVG-002, its novel, inhaled gene therapy for lethal neonatal surfactant protein B deficiency
15 November 2024 - AlveoGene announces that it has been granted a rare paediatric disease designation by the US FDA for AVG-002, its novel inhaled gene therapy for lethal neonatal surfactant protein B deficiency.
Inherited surfactant protein B deficiency is an ultra-rare monogenic cause of fatal respiratory distress syndrome in newborn infants with very limited treatment options.
