The method of our time is to use not one but multiple exploration models.

Marshall McLuhan





BioMarin Achondroplasia Clinical Development Program (BioMarin, November 2018)(pdf)

BioMarin is a global pharmaceutical company that has 20 years of experience in medicine development for rare genetic conditions. BioMarin's investigational medicine for achondroplasia, BMN 111, is currently in the advanced stages of the medicine development process, also known as phase 3 clinical studies. The safety and effectiveness of BMN 111 is currently being investigated in ongoing clinical studies and has not been established. BMN 111 has not been approved for use outside of a clinical study by any country. At the conclusion of the clinical studies, BioMarin can apply for approval of BMN 111 and authorities will evaluate the safety and effectiveness data and determine whether it can be marketed in a given country

Full update here