In that Dept they need Research Scientists Doctors. People that can understand modern science at his/her core and the intent and expectations surrounding spending Billions$$ on a rare DX with the cooperation of Families. That said, make a new FDA department for new Children Technologies.
RIght, WHo knows what the FDA thinks of any of the data. If SRPT posts data as good or better than expected, the FDA might not have the same view.
Everyone with two shares short Posting. It's just too bad we have to put up with this crapola on Yahoo without better BLOCKING ignore user feature.
l that's news after 4 years. Suddenly you "jcordaly" discover this hidden fact NOW and are sharing this explosive tid-bit. Better call the FDA, FCC, BBC and FBI they all seemed to have over looked this drama.
24 weeks showed ADL improvements without Dystrophin showing that much on some kids . However, the parents and kids know it's working. By 48 weeks both ^ADL and ^labs, a bit low if advance with the less muscle to treat. Early reports suggest kids having Positive changes to ALDs on the extended study.
First, it's in the tissue. The tissue is damaged includes major scar tissue and fat. A Small amount of tissue still works if repaired. You will see new protein in whatever undamaged tissue you have. The first kids were so close to failure because they had only a limited available muscle tissue. So low levels would be expected. Earlier kids may have more dystrophin made only because they have more remaining Muscle.
My example, if you had a 1% battery to save your life would you use it? Or be afraid of the call being dropped? These late stage kids have that 1%, and enough to keep them going. Earlier kids would have a bigger muscle battery that could charge let's say to 9%. A winner long term.
Thier are many Monuments overseas, Germany, France Belgium, North Africa. We are coming up on 100 years from WWI. I hope some of you can make it to anyone of those Cemeteries and Battlefields. I would suggest Verdun FR, Bastone or D-day areas. Verdun, walk into an open square miles field and survive. Our soldiers did the improbable with daring and grit. 28,000 in battle and 50,000 or so from flu.