A quick reminder for all of us since I see very little mention of Biomarin's utrophin phase 1 trial.If they get desired results phase 2 could start early next year.We all need to get behind this and do all we can to help speed this one into next level where our son's can potentially take part in their future trial.
Somehow we need to influence biomarin and who better than Pat Furlong can do this for us. We need Biomarin to be as good as Ptc in terms of communication and patient advocacy.Any Ideas please share and lets get behind pushing this one fast.
I listened in to the BIOMARIN Webinar for investors today...here's what was said about BMN-195. And before anyone asks, I'm not sure what kind of increase in utrophin expression one would want to see to translate into strength gains (will check on that), and also keep in mind that this result was in cultured cells.
Hank Fuchs, M.D., Chief Medical Officer of BIOMARIN reported that its utrophin upregulating molecule BMN-195 is currently in a phase I safety study, which was initiated in the first quarter of this year and is scheduled to be completed in the third quarter. He showed data suggesting that in cultured human muscle cells BMN-195 can increase the level of messenger RNA for utrophin by 37% and can increase the level of utrophin protein by 45%.