CFTR-related conditions/cystic fibrosis
CFTR-related conditions, including cystic fibrosis, are a group of disorders that cause the production of abnormally thick and sticky mucus in the lungs and digestive system.
Gaucher disease/GBA-related conditions
Gaucher disease is a metabolic disorder in which the body lacks an enzyme needed to break down a fatty substance called glucocerebroside.
Phenylalanine Hydroxylase Deficiency (PKU)
Phenylalanine hydroxylase deficiency (PHD) is a treatable metabolic disorder in which the body cannot properly break down the amino acid phenylalanine, a building block of protein.
