Browsing by Subject "GENE-THERAPY"

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    ItemOpen Access
    Comparison of US Federal and Foundation Funding of Research for Sickle Cell Disease and Cystic Fibrosis and Factors Associated With Research Productivity.
    (JAMA network open, 2020-03-02) Farooq, Faheem; Mogayzel, Peter J; Lanzkron, Sophie; Haywood, Carlton; Strouse, John J
    Importance:Sickle cell disease (SCD) and cystic fibrosis (CF) are severe autosomal recessive disorders associated with intermittent disease exacerbations that require hospitalizations, progressive chronic organ injury, and substantial premature mortality. Research funding is a limited resource and may contribute to health care disparities, especially for rare diseases that disproportionally affect economically disadvantaged groups. Objective:To compare disease-specific funding between SCD and CF and the association between funding and research productivity. Design, Setting, and Participants:This cross-sectional study examined federal and foundation funding, publications indexed in PubMed, clinical trials registered in ClinicalTrials.gov, and new drug approvals from January 1, 2008, to December 31, 2018, in an estimated US population of approximately 90 000 individuals with SCD and approximately 30 000 individuals with CF. Main Outcomes and Measures:Federal and foundation funding, publications indexed in PubMed, clinical trial registrations, and new drug approvals. Results:From 2008 through 2018, federal funding was greater per person with CF compared with SCD (mean [SD], $2807 [$175] vs $812 [$147]; P < .001). Foundation expenditures were greater for CF than for SCD (mean [SD], $7690 [$3974] vs $102 [$13.7]; P < .001). Significantly more research articles (mean [SD], 1594 [225] vs 926 [157]; P < .001) and US Food and Drug Administration drug approvals (4 vs 1) were found for CF compared with SCD, but the total number of clinical trials was similar (mean [SD], 27.3 [6.9] vs 23.8 [6.3]; P = .22). Conclusions and Relevance:The findings show that disparities in funding between SCD and CF may be associated with decreased research productivity and novel drug development for SCD. Increased federal and foundation funding is needed for SCD and other diseases that disproportionately affect economically disadvantaged groups to address health care disparities.
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    ItemOpen Access
    Inherited Retinal Degenerations: Current Landscape and Knowledge Gaps
    (Translational Vision Science & Technology, 2018-07-18) Duncan, Jacque L; Pierce, Eric A; Laster, Amy M; Daiger, Stephen P; Birch, David G; Ash, John D; Iannaccone, Alessandro; Flannery, John G; Sahel, José A; Zack, Donald J; Zarbin, Marco A; and the Foundation Fighting Blindness Scientific Advisory Board