Fantastically little information provided, yet it's called a "breakthrough!!".
How is the drug dosed? If it's oral, then I'm not surprised. It probably traps the cholesterol in the mouse-model diet before it can even be absorbed.
How is the drug dosed? If it's oral, then I'm not surprised. It probably traps the cholesterol in the mouse-model diet before it can even be absorbed.
They found cyclodextrin and initially tried using it in
oral doses, which is known to be safe. However, the
chemical couldn’t effectively reach the brain that way.
The couple made headlines with their tireless efforts to
get drug companies, the FDA, and doctors to let them try
out intravenous treatments of cyclodextrin for their
twins—and they won. Regular treatments gradually
improved—although didn’t cure—the twins’ conditions.
Cyclodextrin is now in clinical trials to treat other
kids with NPC.When I consume products that contain wheat, I feel sick. When I don't, I feel fine. Sample size n=1, and I am not medically trained. What conclusions, if any, do you think I can draw from my experience?
Science is hard. Control experiments are key.