Treatment
At present, there is no known treatment for achondroplasia, even though the cause of the mutation in the growth factor receptor has been found.
Although used by those without achondroplasia to aid in growth, human growth hormone does not help people with achondroplasia. However, if desired, the controversial surgery of limb-lengthening will lengthen the legs and arms of someone with achondroplasia.
Usually, the best results appear within the first and second year of therapy. After the second year of GH therapy, beneficial bone growth decreases. Therefore, GH therapy is not a satisfactory long term treatment.
Gene based therapy is on the horizon. The USA based company BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) recently announced the initiation of a Phase 1 study in healthy volunteers for BMN-111, an analog of C-type Natriuretic Peptide (CNP), for the treatment of achondroplasia.
Latest research and related efforts are tracked by the non-profit Growing Stronger.
Read more about this topic: Achondroplasia
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—Rutherford Birchard Hayes (1822–1893)
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—Henry David Thoreau (1817–1862)
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—Margaret Mead (1901–1978)