Achondroplasia is a rare genetic disorder of bone growth that causes short-limbed dwarfism. It occurs due to mutations in a single gene called the FGFR3.

Achondroplasia is a rare genetic disorder of bone growth that causes short-limbed dwarfism. It occurs due to mutations in a single gene called the FGFR3.

Achondroplasia is the most common form of skeletal dysplasia or dwarfism, affecting around one in 20,000 babies in Australia. The genetic condition can cause serious medical complications such as ...

BioMarin is a global biotechnology company focused on developing and commercializing therapies for rare genetic diseases. BioMarin specializes in enzyme replacement therapies, gene therapies, and ...