Skip to content

Tag: Rare disease drug

Explore our medication guides and pharmacology articles within this category.

What Type of Drug is PALYNZIQ? An Enzyme Replacement Therapy for PKU

3 min read
Phenylketonuria (PKU) is a rare genetic disease affecting approximately 1 in 12,500 live births in the United States, resulting in the body's inability to properly break down the amino acid phenylalanine. For adults with persistently high and uncontrolled phenylalanine levels despite other treatments, a specialized medication known as PALYNZIQ offers a new therapeutic option by directly addressing this metabolic deficiency.