News

First Mouse Model of AADC Deficiency Developed, Researchers Report

A mouse model of aromatic l-amino acid decarboxylase (AADC) deficiency, carrying a frequent mutation in patients with the condition, has been developed for the first time, according to researchers. Findings from this new model indicate that low levels of the neurotransmitter serotonin are associated with characteristic symptoms. The study, “…

DDC Gene Mutation Study Advances AADC Deficiency Research

Antisense oligonucleotides (ASOs) — short nucleic acid molecules used to modulate protein production — can be used to correct abnormal dopa decarboxylase (DDC) processing in cells from patients with aromatic L-amino acid decarboxylase (AADC) deficiency, a new study reports. The study, “Antisense oligonucleotides modulate dopa decarboxylase function in…

PTC Therapeutics Advances Gene Therapy for AADC Deficiency

PTC Therapeutics plans to request approval for its GT-AADC gene therapy, developed for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency, in the United States. Supported by positive data from clinical studies, the company expects to submit a biologics license application (BLA) with the U.S. Food and Drug Administration…