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Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience

Human Gene Therapy(2023)10.1089/hum.2022.232Source: DataRank Database

Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience is a research paper published in Human Gene Therapy (2023). On theSindex it has a DataRank of 0. It has been cited 10 times.

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0DataRank · unranked

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Open Access10 citations

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›Methodology & internals

datarank_citation_only_1hop_v6· scope data_onlyMethodology

Pipeline:MetadataData-paper checkEnrichmentCitation networkScoring

Enrichment:Funding (2 grants)OA: hybridTopics (14)IDs (PubMed)SDGs (1)

FAIR Checklist

Context only (not used in score)

Findable (1/2)

Has DOI

Accessible (1/2)

Open Access

Interoperable (0/2)

Reusable (0/3)

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Authors (43)

Oleg A. Shchelochkov,Randy J. Chandler ORCID,NIH PaVe-GT Team,Charles P. Venditti ORCID,Anne Pariser ORCID

Live enrichment

PubMed:21097893

National Institutes of Health

Grant: 1ZIATR000281-08

Gene Therapy Platform for Rare Diseases

National Institutes of Health

Grant: 1ZIAHG200318-16

Investigations of Methylmalonic Acidemia and Related Disorders

hybridLicense: cc-by

publisherhttps://doi.org/10.1089/hum.2022.232

publisherhttps://journals.sagepub.com/doi/full-xml/10.1089/hum.2022.232

publisherhttps://journals.sagepub.com/doi/pdf/10.1089/hum.2022.232

repositoryhttps://pmc.ncbi.nlm.nih.gov/articles/PMC10031144/pdf/hum.2022.232.pdf

https://pubmed.ncbi.nlm.nih.gov/36694456

Fields of Study

0301 basic medicine0303 health sciences03 medical and health sciences

Keywords

Propionic AcidemiaOrphan Drug ProductionUnited States Food and Drug AdministrationClinical DevelopmentsGenetic TherapyUnited StatesRare DiseasesNational Institutes of Health (U.S.)HumansChildDrug Approval

Sustainable Development Goals

SDG 3: 3. Good health