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Strengthening health systems for access to gene therapy in rare genetic disorders

Bhatia, Sonal; Le Cam, Yann; Carrion, Juan; Diamond, Lauren; Fennessy, Paul; Gassman, Safiyya; Gutzwiller, Felix; Kagan, Stephen; Pankevich, Diana; Young Maloney, Jennifer; Mahadev, Nitin; Schulz, Martin; Wong-Rieger, Durhane; Morgese, Paolo (2024). Strengthening health systems for access to gene therapy in rare genetic disorders. Molecular Therapy - Methods & Clinical Development, 32(2):101220.



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Item Type:Journal Article, refereed, original work
Communities & Collections:04 Faculty of Medicine > Epidemiology, Biostatistics and Prevention Institute (EBPI)
Dewey Decimal Classification:610 Medicine & health
Scopus Subject Areas:Life Sciences > Molecular Medicine
Life Sciences > Molecular Biology
Life Sciences > Genetics
Uncontrolled Keywords:Genetics, Molecular Biology, Molecular Medicine
Date:13 June 2024
Deposited On:12 Apr 2024 14:33
Last Modified:13 Apr 2024 20:00
Publisher:Cell Press (Elsevier)
OA Status:Gold
Free access at:Publisher DOI. An embargo period may apply.
Publisher DOI:https://doi.org/10.1016/j.omtm.2024.101220
Official URL:https://www.sciencedirect.com/science/article/pii/S2329050124000366
PubMed ID:38516694
Project Information:
  • : FunderPfizer
  • : Grant ID
  • : Project Title
  • Content: Published Version
  • Language: English
  • Licence: Creative Commons: Attribution 4.0 International (CC BY 4.0)