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Jilly_in_VA

(13,594 posts)
Mon Nov 24, 2025, 01:36 PM 21 hrs ago

Boy with rare condition amazes doctors after world-first gene therapy

A three-year-old boy has astounded doctors with his progress after becoming the first person in the world with his devastating disease to receive a ground-breaking gene therapy.

Oliver Chu has a rare, inherited condition called Hunter syndrome - or MPSII - which causes progressive damage to the body and brain.

In the most severe cases, patients with the disease usually die before the age of 20. The effects are sometimes described as a type of childhood dementia.

Due to a faulty gene, before the treatment Oliver was unable to produce an enzyme crucial for keeping cells healthy.

In a world first, medical staff in Manchester have tried to halt the disease by altering Oliver's cells using gene therapy.
(snip)

A year after starting the treatment, Oliver now appears to be developing normally.

https://www.bbc.com/news/articles/c5y0y56x6veo

This kind of thing would not be possible right now in the US, thanks to RFK Jr.

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Boy with rare condition amazes doctors after world-first gene therapy (Original Post) Jilly_in_VA 21 hrs ago OP
"Healthcare systems of different countries! What are y'all working on?" ck4829 21 hrs ago #1
Truly an event to be sincerely grateful for! SheltieLover 21 hrs ago #2
It would here timms139 20 hrs ago #3

ck4829

(37,278 posts)
1. "Healthcare systems of different countries! What are y'all working on?"
Mon Nov 24, 2025, 01:43 PM
21 hrs ago

UK healthcare system: We found a way to treat Hunter syndrome!

OK... US healthcare system, how about you?

US healthcare system: Well we found a way to see how many steps a patient takes in the hospital so we can add it to their final medical bill! Take that UK!

timms139

(455 posts)
3. It would here
Mon Nov 24, 2025, 02:40 PM
20 hrs ago

most likely cost a millon or more so probably out of reach unless those here could travel to the UK. . We all see what they do with the price of the life saving drug insulin here .

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