BioMarin Pharmaceutical Inc. has advancements in phenylketonuria treatment. Click to learn how Palynziq's success could ...

The incidence of hypochondroplasia is not known, but it has been estimated to be about as common as achondroplasia, which is seen in around 1 in 15,000 to 40,000 births. BioMarin has said that ...

Although achondroplasia and hypochondroplasia are considered distinct clinical diagnoses, they share a significant amount of overlapping features, according to a poster presented at the 2025 American ...

BioMarin Pharmaceutical Inc.’s BMRN share price has dipped by 10.70%, which has investors questioning if this is right time ...

Three INDs cleared by US FDA for TYRA's proprietary precision small molecules - - TYRA-300 to be evaluated in three Phase 2 studies: SURF ...

The company’s products under development include BMN 333, a longer-acting CNP for the treatment of multiple growth disorders, such as achondroplasia and hypochondroplasia; BMN 349, an oral ...

TYRA-300 is an investigational, oral, FGFR3-selective inhibitor currently in development for the treatment of cancer and skeletal dysplasia, including achondroplasia and hypochondroplasia.

TransCon CNP is an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly and designed to treat individuals with achondroplasia by providing continuous exposure of ...