{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["44(1)"],"submitter":["Zhang R"],"funding":["PTC Therapeutics"],"pubmed_abstract":["<h4>Background</h4>Recently, the gene therapy eladocagene exuparvovec received accelerated approval from the US Food and Drug Administration (as eladocagene exuparvovec-tneq) for treatment of aromatic L-amino acid decarboxylase deficiency (AADCd), a rare, infantile-onset disorder characterized by developmental delays.<h4>Objectives</h4>To conduct a US, modified societal perspective cost-utility analysis comparing eladocagene exuparvovec versus best supportive care (BSC).<h4>Methods</h4>Multistate survival modeling was implemented tracking disease progression from a \"no motor function\" health state to achievement of motor-function improvements, measured by: (1) multiples of the meaningful score difference (MSD) of the Peabody Developmental Motor Scales-Second Edition (PDMS-2) total score an"],"journal":["PharmacoEconomics"],"pagination":["25-41"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC12799622"],"repository":["biostudies-literature"],"pubmed_title":["Eladocagene Exuparvovec for the Treatment of Aromatic L-Amino Acid Decarboxylase Deficiency (AADCd): An Economic Evaluation from a US Perspective."],"pmcid":["PMC12799622"],"pubmed_authors":["O'Connell T","Tomazos I","Monteleone B","Teng Y","Zhang R","Hwu PW","Castellano P"],"additional_accession":[]},"is_claimable":false,"name":"Eladocagene Exuparvovec for the Treatment of Aromatic L-Amino Acid Decarboxylase Deficiency (AADCd): An Economic Evaluation from a US Perspective.","description":"<h4>Background</h4>Recently, the gene therapy eladocagene exuparvovec received accelerated approval from the US Food and Drug Administration (as eladocagene exuparvovec-tneq) for treatment of aromatic L-amino acid decarboxylase deficiency (AADCd), a rare, infantile-onset disorder characterized by developmental delays.<h4>Objectives</h4>To conduct a US, modified societal perspective cost-utility analysis comparing eladocagene exuparvovec versus best supportive care (BSC).<h4>Methods</h4>Multistate survival modeling was implemented tracking disease progression from a \"no motor function\" health state to achievement of motor-function improvements, measured by: (1) multiples of the meaningful score difference (MSD) of the Peabody Developmental Motor Scales-Second Edition (PDMS-2) total score an","dates":{"release":"2026-01-01T00:00:00Z","publication":"2026 Jan","modification":"2026-06-06T13:18:33.727Z","creation":"2026-05-31T03:07:26.887Z"},"accession":"S-EPMC12799622","cross_references":{"pubmed":["40991122"],"doi":["10.1007/s40273-025-01542-8"]}}