Securitization as a means to pay for cell and gene therapies for orphan diseases: a simulation study. [PDF]
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External controls for rare disease drug development: Lessons for emerging and advanced therapeutic modalities. [PDF]
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Frontiers in Cellular Neuroscience, 2022 Wilfried Rossoll, Ravindra N. Singh
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Broadening applications for intrathecal gene therapy: a case for lysosomal storage diseases. [PDF]
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How much do ex-US revenues make a difference for pharmaceutical investment returns? [PDF]
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