Posted AT 12:56 PM EDT on 13/05/08
Gene trial shows promise in rare disease
WASHINGTON — An experimental gene therapy treatment appears to have helped eight children with a rare and incurable neurological disorder, although it may have been responsible for the death of one, researchers reported on Tuesday.
They said the treatment appeared safe and effective enough to try in more children with late infantile neuronal ceroidlipofuscinosis (LINCL), a form of deadly Batten disease.
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