U.S. Food and Drug Administration

FDA Greenlights Aqneursa for Niemann-Pick Disease Type C, Marking a Breakthrough in Rare Disease Treatment

WASHINGTON D.C. — The U.S. Food and Drug Administration (FDA) has made a landmark decision to approve Aqneursa (levacetylleucine) for the treatment of neurological symptoms linked to Niemann-Pick disease type C …

FDA Greenlights Aqneursa for Niemann-Pick Disease Type C, Marking a Breakthrough in Rare Disease Treatment Read More