Childhood Degenerative & Metabolic Disorders News


Mar 23, 2020
New brain disorder discovery
A study has uncovered a new neurodegenerative disorder in which children experience developmental regression and severe epilepsy.
Jan 10, 2019
Researchers identify a new leukodystrophy in children and its potential cure
The gene that causes the disease is called DEGS1 and its defect may be counteracted with fingolimod, a drug currently used to treat multiple sclerosis.
Jul 16, 2018
Fetal gene therapy prevents fatal neurodegenerative disease
A fatal neurodegenerative condition known as Gaucher disease can be prevented in mice following fetal gene therapy.