The few notable exceptions include Biomarin’s drug candidate for achondroplasia (ACH), a C-type natriuretic peptide (CNP) analogue PG-CNP37 (BMN-111) [3], bisphosphonate treatment for OI [4] and fibrous dysplasia [5], enzyme replacement in lysosomal storage diseases [6] or hematopoietic stem cell transplantation for infantile osteopetrosis [7]. The gene discussed is NPPC; the disease is achondroplasia.