The R&D head is looking for "clever science," which Friberg said is readily apparent in BioMarin’s recent research pact with CAMP4 Therapeutics. The partners are using CAMP4’s regulatory RNA platform ...

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.

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 ...

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 ...

I look forward to continuing to work with this stellar team to serve patients with genetic disease in 2025 ... Infigratinib – FGFR1-3 inhibitor for achondroplasia and hypochondroplasia ...