Nature Medicine

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Barak, T., Ristori, E., Ercan-Sencicek, A. G., Miyagishima, D. F., Nelson-Williams, C., Dong, W., Jin, S. C., Prendergast, A., Armero, W., Henegariu, O., Erson-Omay, E. Z., Harmancı, A. S., Guy, M., Gültekin, B., Kilic, D., Rai, D. K., Goc, N., Aguilera, S. M., Gülez, B., … Günel, M. (2021). PPIL4 is essential for brain angiogenesis and implicated in intracranial aneurysms in humans. Nature Medicine, 27(12), 2165–2175. https://doi.org/10.1038/s41591-021-01572-7
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Gotlib, J., Reiter, A., Radia, D. H., Deininger, M. W., George, T. I., Panse, J., Vannucchi, A. M., Platzbecker, U., Alvarez-Twose, I., Mital, A., Hermine, O., Dybedal, I., Hexner, E. O., Hicks, L. K., Span, L., Mesa, R., Bose, P., Pettit, K. M., Heaney, M. L., … DeAngelo, D. J. (2021). Efficacy and safety of avapritinib in advanced systemic mastocytosis: interim analysis of the phase 2 PATHFINDER trial. Nature Medicine, 27(12), 2192–2199. https://doi.org/10.1038/s41591-021-01539-8
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Thomsen, G., Burghes, A. H. M., Hsieh, C., Do, J., Chu, B. T. T., Perry, S., Barkho, B., Kaufmann, P., Sproule, D. M., Feltner, D. E., Chung, W. K., McGovern, V. L., Hevner, R. F., Conces, M., Pierson, C. R., Scoto, M., Muntoni, F., Mendell, J. R., & Foust, K. D. (2021). Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue. Nature Medicine, 27(10), 1701–1711. https://doi.org/10.1038/s41591-021-01483-7
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Ratziu, V., de Guevara, L., Safadi, R., Poordad, F., Fuster, F., Flores-Figueroa, J., Arrese, M., Fracanzani, A. L., Ben Bashat, D., Lackner, K., Gorfine, T., Kadosh, S., Oren, R., Halperin, M., Hayardeny, L., Loomba, R., Friedman, S., Abdelmalek, M., … Sanyal, A. J. (2021). Aramchol in patients with nonalcoholic steatohepatitis: a randomized, double-blind, placebo-controlled phase 2b trial. Nature Medicine, 27(10), 1825–1835. https://doi.org/10.1038/s41591-021-01495-3
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