Sad situation, but the parents were not even allowed to try facilities in Italy. I thought I read they had been ‘given permission’ to transfer their daughter to Rome. I guess I mixed up the facts.
“Permission by The Government??!!” American parents should not gloat too much that ‘this could never happen here!’.
Look at the M.A.I.D. system in Canada right now, only a hoot and a holler away from our northern states.
The UK government cheered on murder in Ukraine on the battlefield so why not some killing on “The Home Front”.
If I were POTUS this would not stand there was a Suez Crisis when Eisenhower put the British government in its place.
It must be replayed in cases like this.
There is an experimental drug in early phase 2 clinical trials that might be able to treat Leigh's disease. The primary purpose of phase 2 is to see if the drug "works." Phase 2 studies are typically very small. Mitochondrial Disease Life Expectancy.
There are 14 studies registered at www.clinicaltrials.gov for one of the drugs mentioned in this article, EPI-743. Only two of them were focused on Leigh's disease patients. Mitochondrial diseases EPI743 search results.
When I looked for clinical trials of Leigh's disease, I found 17 studies registered. Mitochondrial disease Leigh search results.
Of these studies, three are registries, meaning that they are aimed at locating patients and recording their symptoms in order to build a more complete clinical profile of the disease. One was withdrawn for reason of IND withdrawal (meaning that the FDA pulled the "investigational new drug" designation needed to perform clinical trials). Another was terminated for lack of efficacy of the drug being tested. There were only three drugs being tested in the studies of potential treatments, one of them being the drug with lack of efficacy.
None of the drugs being tested would cure the disease. They are intended to try to chemically enhance mitochondrial function to overcome the defect in the electron transport chain. So far, results are not promising.
Thus, the only option possible would have been to enroll the child in a phase 2 early drug study. Maybe that's what the hospital in Italy wanted to do; I don't know. Would enrolling the child in an experimental study of a drug to see if/how well it works be of psychological benefit to the parents? The child's disease is still fatal. Perhaps enrolling her in a clinical trial would have given the parents the feeling that they did everything possible, which would allow them to accept the death more peacefully? We will never know.
Jeb moved to England? Who knew?