From
November 6, 2009
A devastating brain disease that kills boys by the time of adolescence has been stopped in its tracks by a gene therapy that raises hope for thousands of families.
The treatment has halted the development of adrenoleukodystrophy (ALD) — the genetic disorder featured in the film Lorenzo’s Oil — in two Spanish boys aged 7 who would otherwise have suffered progressive and ultimately fatal brain damage. A third boy has also been treated, but too recently for doctors to know whether it has been successful.
