26 Nov This lifesaving treatment was designed for one. Could it be the future of medical care?

By Karen Weintraub, USA Today

Imagine you or your child gets a diagnosis so rare no one else on Earth is known to have it.

Doctors can do nothing but predict a terrible downward spiral followed by death.

That’s the situation Luke Rosen and Sally Jackson found themselves in when their daughter Susannah was diagnosed in 2016 with an ultra-rare genetic condition.

They were told their daughter, who has a mutation in a gene called KIF1A, had about five years before her condition would begin affecting her beyond repair. At year six, they met a man named Stanley Crooke who promised to develop a medication just for Susannah.

“Right on the brink of hopelessness, we knew that Susannah could possibly and hopefully have a different life,” said Rosen, a firefighter and former actor in Long Island, New York.

Now, after a year of an experimental treatment, the results are even better than they could have wished for. Read more…