Most notably, the studies are based on transient transfection of wild - type and mutant gene constructs into cultured cells or into zebrafish embryos and are unlikely to reproduce the specific mutant gene dose that is present in heterozygous mutant cells in FOP patients. (jci.org)
«The implications for targeted clinical trials and for compassionate clinical use of HIF - 1α inhibitors in the treatment of FOP flare - ups are promising, however we need more data on dosing, duration, timing, rebound, resistance and long - term safety,» Pignolo said. (sciencedaily.com)
Our concern was that treating children with FOP with palovarotene would synergize to further impact their overall growth, potentially leading to additional health complications. (sciencedaily.com)