A better pathophysiological understanding of the disease, as well as therapeutic progress and implementation of a generalized neonatal screening targeting newborns at risk, currently enables more than 90% of affected children to reach adulthood
A betterpathophysiological understanding of the disease, as wellas therapeutic progress and implementation of a generalizedneonatal screening targeting newborns at risk, currentlyenables more than 90% of affected children to reach adulthood