Achondroplasia & FGFR3
This page was produced as an assignment for Genetics 677 at UW-Madison, Spring 2010
Chemical Genetics
Searches on Drugbank and Entrez revealed that there are no chemical compounds that target FGFR3 in order to treat achondroplasia. Other mutations in FGFR3 can cause certain forms of cancer, and there are drugs like Palifermin that target the receptor to treat these. Perhaps in the future it will be found that these can also be effective on cells with the mutation that causes achondroplasia.
Unfortunately, children are born already exhibiting the symptoms of achondroplasia, as can be seen in the image of the little girl on the Homepage of this website. So, I would speculate that any drug that would truly have an effect on altering bone development may have to be given when the child is relatively young. This seems like a complicated solution, since exposing a developing child to a chemical compound could have profound effects on other parts of his development.
Unfortunately, children are born already exhibiting the symptoms of achondroplasia, as can be seen in the image of the little girl on the Homepage of this website. So, I would speculate that any drug that would truly have an effect on altering bone development may have to be given when the child is relatively young. This seems like a complicated solution, since exposing a developing child to a chemical compound could have profound effects on other parts of his development.
