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aromatic amino acid decarboxylase deficiency
This page shows the latest aromatic amino acid decarboxylase deficiency news and features for those working in and with pharma, biotech and healthcare.
Positive CHMP opinion issued for PTC Therapeutics’ Upstaza
The therapy will be the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency for patients who are 18 months and older, and will be the first ... There are currently no approved disease-modifying treatments
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Researchers publish promising results for ultra-rare disease gene therapy method
Gene therapy method evaluated in seven children with aromatic amino acid decarboxylase deficiency. ... Researchers have published some promising results for a new gene therapy method that may offer hope for children born with the ultra-rare genetic
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