Tomas was diagnosed with ornithine transcarbamylase (OTC) deficiency at Great Ormond Street Hospital for Children last year. He took part in the OTC-HOPE clinical trial, where he received the investigational gene therapy, ECUR-506.
ECUR-506 is designed as a one-time treatment to permanently insert a functional OTC gene (see BioNews 1272).
The initial data was presented at the Annual Clinical Genetics Meeting in Los Angeles, California.
Read more in the Independent.