A Comparative Effectiveness Study of Newborn Screening Methods for Four Lysosomal Storage Disorders
Lysosomal storage disorders (LSDs) are a group of more than 50 inherited metabolic disorders where enzyme deficiency inhibits the ability of the lysosomes present in each of the body’s cells to function normally. Recent discoveries of new therapies coupled with novel screening methods has led to the adoption of newborn screening for several LSDs around…
Read more
Recent Comments