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.
while being agnostic for the FGFR3 gatekeeper mutations. TYRA-300 is in development for the treatment of cancer in the SURF301 Phase 1/2 study and for achondroplasia in the BEACH301 Phase 2 study.
I was born with achondroplasia, the most common type of dwarfism. My parents are both of average height, and my brother is over 6 feet tall. But through a fluke of genetic mutations, I was given ...