{"doi":"10.1007/s40265-022-01775-3","title":"Eladocagene Exuparvovec: First Approval","abstract":null,"journal":"Drugs","year":2022,"id":650946,"datarank":0.6284482113039639,"base_score":4.189654742026425,"endowment":4.189654742026425,"self_citation_contribution":0.6284482113039639,"citation_network_contribution":0.0,"self_endowment_contribution":0.6284482113039639,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":65,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":null,"is_data_producer":false,"deposit_databanks":null,"is_oa":false,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":null,"fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":1559664,"name":"Susan J. Keam","orcid":null,"position":0,"is_corresponding":false}],"reference_count":0,"raw_metadata":{"has_enrichment":true,"resolved":true,"title":"Eladocagene Exuparvovec: First Approval","abstract":"Eladocagene exuparvovec (Upstaza™) is a gene therapy developed by PTC Therapeutics for the treatment of human aromatic L-amino acid decarboxylase (AADC) deficiency. Eladocagene exuparvovec comprises an adeno-associated virus vector that delivers the dopa decarboxylase (DDC) gene, the gene for human AADC. Eladocagene exuparvovec was approved in July 2022 in the EU for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of AADC deficiency with a severe phenotype (i.e. patients who cannot sit, stand or walk). This article summarizes the milestones in the development of eladocagene exuparvovec leading to this first approval for the treatment of patients aged 18 months and older with AADC deficiency.","is_dataset_classified":null,"base_score":4.189654742026425,"endowment":4.189654742026425,"datacite_reuse_total":0,"file_count":0,"downloads":0,"views":0,"has_version_chain":false,"is_dataset":false,"is_oa":false,"pmid":"36103022","pmcid":null,"openalex_id":"https://openalex.org/W4295654927","authors":[],"funders":[],"total_grants":0,"fwci":7.8203,"citation_percentile":0.98589504,"influential_citations":0,"citation_trend":[{"year":2022,"count":1},{"year":2023,"count":16},{"year":2024,"count":29},{"year":2025,"count":17},{"year":2026,"count":2}],"oa_status":"green","license":"cc-by-nc","oa_locations":[{"url":"https://figshare.com/articles/online_resource/Eladocagene_Exuparvovec_First_Approval/20761024","host_type":"repository"},{"url":"https://figshare.com/articles/online_resource/Eladocagene_Exuparvovec_First_Approval/20761024","host_type":"repository"},{"url":"https://link.springer.com/content/pdf/10.1007/s40265-022-01775-3.pdf","host_type":"publisher"},{"url":"https://link.springer.com/article/10.1007/s40265-022-01775-3/fulltext.html","host_type":"publisher"},{"url":"https://doi.org/10.1007/s40265-022-01775-3","host_type":"journal"},{"url":"https://pubmed.ncbi.nlm.nih.gov/36103022","host_type":"repository"},{"url":"https://doi.org/10.6084/m9.figshare.20761024.v1","host_type":"repository"},{"url":"https://figshare.com/articles/online_resource/Eladocagene_Exuparvovec_First_Approval/20761024/1/files/37420288.pdf","host_type":"Unpaywall"}],"fields_of_study":["Metabolism and Genetic Disorders","Adipose Tissue and Metabolism","Biotin and Related Studies","Amino Acid Metabolism, Inborn Errors","Amino Acids","Aromatic-L-Amino-Acid Decarboxylases","Dopa Decarboxylase","Humans","Aromatic amino acid decarboxylase deficiency"],"mesh_terms":["Amino Acid Metabolism, Inborn Errors","Amino Acids","Aromatic-L-Amino-Acid Decarboxylases","Dopa Decarboxylase","Humans"],"keywords":["Medicine","Genetic enhancement","Aromatic L-amino acid decarboxylase","Adeno-associated virus","Pharmacotherapy","Clinical trial","Gene","Pharmacology","Internal medicine","Pediatrics","Vector (molecular biology)","Recombinant DNA","Genetics","Dopamine","Biology"],"sdg_mappings":[{"sdg_number":0,"sdg_label":"Good health and well-being"}],"linked_datasets":[],"clinical_trials":[],"software_tools":[],"database_accessions":[],"source":"live","citation_network_status":"fetched"},"created_at":"2026-08-10T06:23:19.696127Z","pmid":null,"pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}