Author Archives: Joana Carvalho

International Research Group Works to Increase Knowledge, Awareness of Neurotransmitter Disorders

Since its founding in 2013, the International Working Group on Neurotransmitter related Disorders (iNTD) has been building a network and patient registry aimed at promoting knowledge and awareness among clinicians on rare neurotransmitter disorders, including aromatic l-amino acid decarboxylase (AADC) deficiency. Over the past several years, the collaboration has expanded…

Agilis Biotherapeutics’ AADC Gene Therapy Ready for Submission to FDA

Only 18 months after its initial licensing, Agilis Biotherapeutics‘ gene therapy for aromatic l-amino acid decarboxylase (AADC) deficiency has been deemed ready for submission to the U.S. Food and Drug Administration (FDA). Given its rare disease status, with roughly 120 cases of AADC deficiency described to date, this is a “remarkable…