Posted on Feb 19, 2023

1st UK child to receive gene therapy for fatal genetic disorder is now 'happy and healthy'

Lt Col Charlie Brown

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A 19-month-old girl named Teddi recently became the first child in the U.K. outside a clinical trial to receive a new gene therapy for metachromatic leukodystrophy (MLD), a fatal genetic disorder, the National Health Service (NHS) announced(opens in new tab).

Roughly six months out from treatment, "Teddi is a happy and healthy toddler showing no signs of the devastating disease she was born with," the NHS statement reads.

1st UK child to receive gene therapy for fatal genetic disorder is now 'happy and healthy'

Posted from livescience.com

Posted in these groups: England

Edited 3 y ago

Posted 3 y ago

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Lt Col Charlie Brown

Posted 3 y ago

I expect to see more of this with respect to fatal genetic flaws that are only single flaws...the more complicated issues will harder to do. An example would be Tay-Sachs
https://www.mayoclinic.org/diseases-conditions/tay-sachs-disease/symptoms-causes/syc-20378190