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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…