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Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment.
Ramos DM, d'Ydewalle C, Gabbeta V, Dakka A, Klein SK, Norris DA, Matson J, Taylor SJ, Zaworski PG, Prior TW, Snyder PJ, Valdivia D, Hatem CL, Waters I, Gupte N, Swoboda KJ, Rigo F, Bennett CF, Naryshkin N, Paushkin S, Crawford TO, Sumner CJ. Ramos DM, et al. J Clin Invest. 2019 Nov 1;129(11):4817-4831. doi: 10.1172/JCI124120. J Clin Invest. 2019. PMID: 31589162 Free PMC article. Clinical Trial.