Since the discovery of induced Pluripotent Stem cells, this kind of cell, which can become most types of cells in the organism, have been used in a variety of research programs. Now it's being used in drug discovery for achondroplasia, in this case, to test statins for their capacity of inducing differentiation of these cells into cartilage cells.

There's News on the Phase 2 clinical trial for BMN-111 from BioMarin. The company is enrolling more children into the clinical trial, which is an indication that the data for the Phase 2 is favorable and with reduced side effects.