Gene therapy, if administered early, preserves motor and cognitive functions. Newborn screening is crucial but not yet widespread. The study is the result of 20 years of research at the San ...
If administered early, gene therapy has the potential to change the medical history of children born with metachromatic leukodystrophy (MLD), a rare and lethal neurodegenerative disease of genetic ...
HUNTSVILLE, Ala. (WHNT) — Lilly Lou Gray is a three-and-a-half-year student at Epworth United Methodist Church Pre School and Kindergarten. She was born with a rare genetic disorder called ...
If administered early, gene therapy has the potential to change the medical history of children born with metachromatic leukodystrophy (MLD), a rare and lethal neurodegenerative disease of genetic ...
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