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Amazing medical treatment for rare disease

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Rosie51 Wed 15-Feb-23 10:32:02

www.bbc.co.uk/news/health-64629680

I wondered whether this should go in "Health" but I think it deserves more exposure, and it is very good news!.

Such joy and sadness for this family, but what hope for the future and the scientists believe this treatment could be used for other conditions.

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sodapop Wed 15-Feb-23 12:41:40

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.

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62Granny Wed 15-Feb-23 12:52:10

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.

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Rosie51 Wed 15-Feb-23 15:48:12

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.

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