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2019 3
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Page 1
Impaired prenatal motor axon development necessitates early therapeutic intervention in severe SMA.
Kong L, Valdivia DO, Simon CM, Hassinan CW, Delestrée N, Ramos DM, Park JH, Pilato CM, Xu X, Crowder M, Grzyb CC, King ZA, Petrillo M, Swoboda KJ, Davis C, Lutz CM, Stephan AH, Zhao X, Weetall M, Naryshkin NA, Crawford TO, Mentis GZ, Sumner CJ. Kong L, et al. Sci Transl Med. 2021 Jan 27;13(578):eabb6871. doi: 10.1126/scitranslmed.abb6871. Sci Transl Med. 2021. PMID: 33504650 Free PMC article.
TRPV4 Antagonism Prevents Mechanically Induced Myotonia.
Dupont C, Novak K, Denman K, Myers JH, Sullivan JM, Walker PV 2nd, Brown NL, Ladle DR, Bogdanik L, Lutz CM, A Voss A, Sumner CJ, Rich MM. Dupont C, et al. Ann Neurol. 2020 Aug;88(2):297-308. doi: 10.1002/ana.25780. Epub 2020 Jun 22. Ann Neurol. 2020. PMID: 32418267 Free PMC article.
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.