Amazing medical treatment for rare disease

At the bottom of the article I linked to, there is a boy who was part of the trials in Italy

It is too early to tell, but the signs are good. Several children from the UK were involved in clinical trials of Libmeldy in Milan, before it became a licensed treatment.
Joe Elson, who is 12, had his gene therapy in Italy in 2014. Nine years on, he is completely healthy and doing well at school

He lost his older sister for whom it was too late, but the hope it can bring for those diagnosed younger is wonderful.

A very bittersweet pill for the family. And the "if only feelings " they must have. Let's hope the treatment will be a success for the younger child. It is only with breakthroughs like this in diagnosis and treatment that things move forward.

It is indeed a bittersweet story. The story was shown on breakfast TV this morning.
The consultant involved said he was in awe of the family and the way they were handling things.
In the darkest days there can be hope.