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- W4313887753 abstract "Single-arm trials are often the option for studying cell and gene therapies (CGTs) in rare diseases where there are substantial unmet medical needs with a limited number of patients, or drug administration involves invasive procedures. Natural history studies play critical roles in identifying the target patient population and selecting clinical outcomes or biomarkers, and the source of alternative comparator groups can serve as a potential external control in clinical trials. The development of Zolgensma, a gene therapy approved for the rare disease of spinal muscular atrophy, can be used as a case study for the illustration of the potential roles of natural history studies in the development of CGTs." @default.
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- W4313887753 date "2023-01-09" @default.
- W4313887753 modified "2023-09-23" @default.
- W4313887753 title "Case Studies of Rare Disease Drug Development" @default.
- W4313887753 doi "https://doi.org/10.1201/9781003080954-8" @default.
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