Eladocagene exuparvovec
Published , updated
Unassigned
New Medicines
Aromatic L-amino acid decarboxylase (AADC) deficiency
Information
Advanced therapy medicinal product (ATMP)
PTC Therapeutics
PTC Therapeutics
Development and Regulatory status
None
Phase II Clinical Trials
Phase II Clinical Trials
Yes
Yes
Category
Gene therapy correcting the DDC gene defect resulting in a sustained increase in production of AADC in target areas of the CNS thus increasing the production of critical neurotransmitters
A very rare, severe, genetic neurometabolic disorder associated with clinical manifestations related to underproduction of serotonin and dopamine, mainly hypotonia, hypokinesia, ptosis oculogyric crises, and signs of autonomic dysfunction. Prevalence is <1 in 1 million [1].
Aromatic L-amino acid decarboxylase (AADC) deficiency
Intracerebral