This is a terribly sad story. The little boy needs a not-yet-FDA-approved drug. The company got about $72 million to develop such a drug, but it has not yet been FDA-approved. Previously, they have made some "emergency" dispensations of the drug. Now they say they can't afford to do that any more. They have to devote their resources to getting the drug FDA-approved. It doesn't say how many "emergency" dispensations have been made in the past. The mother of the boy says that there are "hundreds or thousands" of individuals who need the drug to survive.

While there is no one who could not empathize with the mother's plight, there is also the story behind that about how much it costs to bring a drug to market; and the litigation that could ensue, against the company in question, if someone who gets the non-FDA-approved drug were to have a serious adverse reaction.

While $72 million is a lot of money, it may not be much in terms of the cost to bring such a drug to market. I don't know what that cost might be, but it's commonplace for it to take years and much paperwork & testing to approval. A friend who worked for Pfizer several years ago mentioned literally a pallet of paper for the background work on one of their new drugs there at the time. There has to be a way to make this approval process better.

Meanwhile, researchers have come up with a simple blood test to predict onset of Alzheimer's 3 years in advance of its symptoms! It was a 5-year study, and 1/2 way through the study they compared blood test results when they compared 53 patients with onset of symptoms to those not showing symptoms. It was a simple difference in blood lipids. So, a blood test every 2-3 years could ultimately identify those who may need to prepare for the onset of symptoms. That might then lead to finding ways to defer the onset of symptoms for a longer period?