Metabolite repair enzymes restore normal function by correcting inadvertent side‐products that arise during metabolism. These enzymes serve a critical role in maintaining cellular homeostasis, ...

PHILADELPHIA, April 28, 2026--iECURE to Present Preliminary Clinical Data from Ongoing OTC-HOPE Trial of ECUR-506 in Neonatal ...

Please provide your email address to receive an email when new articles are posted on . The FDA has approved an adeno-associated virus vector-based gene therapy to treat both adult and pediatric ...

Aromatic amino acid decarboxylase (AADC) deficiency is a rare, autosomal recessively inherited neurometabolic disorder characterised by markedly reduced synthesis of key monoamine neurotransmitters ...

Dr. Rebecca Ahrens-Nicklas is an Assistant Professor of Pediatrics in the Division of Human Genetics and Metabolism at Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania.

Neuroplasticity and development have traditionally focused on synaptic mechanisms as the primary substrates of circuit remodeling. However, recent advances ...