US EditionBaby given new gene therapy in groundbreaking trial
- A fourteen-month-old baby boy named Tomas is the first patient to receive new gene therapy for ornithine transcarbamylase deficiency, showing significant progress after treatment.
- Tomas was diagnosed with ornithine transcarbamylase deficiency at a few weeks old and was at risk of life-threatening complications without treatment.
- The gene therapy trial at Great Ormond Street Hospital in London may pave the way for treating severe liver genetic diseases in babies.
- Dr. Julien Baruteau reports that Tomas no longer requires standard treatment and has made remarkable improvements in his eating and health.
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Baby given new gene therapy in groundbreaking trial
It is hoped that the new therapy will work as well as a liver transplant for some patients.
·London, United Kingdom
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