Treatment with AGIL-AADC gene therapy induces sustained improvements in motor, cognitive, and language measures in children with aromatic L-amino acid decarboxylase (AADC) deficiency for up to five years, researchers report. Their work, “AGIL-AADC Gene Therapy Results in Sustained Improvements in Motor and Developmental Milestones Over 5 Years in…
Gene therapy has demonstrated the ability to improve motor function in movement disorders, including ones triggered by aromatic L-amino acid decarboxylase (AADC) deficiency, a study reports. According to the researchers, better motor performance observed in children with AADC deficiency after undergoing gene therapy may be associated with improvements in brain…
Drugs used in Parkinson’s disease were seen to increase muscle tone and reduce eyelid droopiness and involuntary upward deviation of the eyes in a boy with aromatic L-amino acid decarboxylase (AADC) deficiency. The case report, “Feeding difficulty and developmental delay for 8 months and nystagmus for…
Microstructural changes were observed in brain regions related to language and motor function in children with aromatic L-amino acid decarboxylase (AADC) deficiency, suggesting that this enzyme shortage can influence brain development, researchers have found. Their study, “Microstructural Changes of Brain in Patients with Aromatic L-Amino Acid…
A generalized dopamine deficiency in aromatic L-acid decarboxylase (AADC) deficiency causes some neurons to be overly active, which may contribute to the motor dysfunction associated with the condition, researchers report. A gene therapy that restores brain dopamine levels seems to repair these abnormalities, their study shows. The study, “Electrical…
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