- Argentinian couple, Natalia and Juan Lovato, moved to Ohio from Guatemala for their son Ciro to join a gene therapy trial for Canavan disease
- Canavan disease is a rare genetic disorder causing progressive brain atrophy and affecting fewer than 50,000 children in the US
- Gene therapy trial in Ohio involves introducing a modified virus to deliver DNA into brain cells to improve myelin function
- Children with Canavan disease typically have a life expectancy of about 10 years, but gene therapy trials have shown encouraging results in stabilizing health
- Challenges include the high cost of gene therapy, selective trial eligibility criteria, and the need for ongoing physical therapy to complement the treatment
https://www.theguardian.com/us-news/article/2024/may/18/argentina-toddler-canavan-disease-trial
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