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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. Among authors: valdivia do. Sci Transl Med. 2021 Jan 27;13(578):eabb6871. doi: 10.1126/scitranslmed.abb6871. Sci Transl Med. 2021. PMID: 33504650 Free PMC article.
Astrocytes influence the severity of spinal muscular atrophy.
Rindt H, Feng Z, Mazzasette C, Glascock JJ, Valdivia D, Pyles N, Crawford TO, Swoboda KJ, Patitucci TN, Ebert AD, Sumner CJ, Ko CP, Lorson CL. Rindt H, et al. Hum Mol Genet. 2015 Jul 15;24(14):4094-102. doi: 10.1093/hmg/ddv148. Epub 2015 Apr 24. Hum Mol Genet. 2015. PMID: 25911676 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.
Boosting neuregulin 1 type-III expression hastens SMA motor axon maturation.
Kong L, Hassinan CW, Gerstner F, Buettner JM, Petigrow JB, Valdivia DO, Chan-Cortés MH, Mistri A, Cao A, McGaugh SA, Denton M, Brown S, Ross J, Schwab MH, Simon CM, Sumner CJ. Kong L, et al. Among authors: valdivia do. Acta Neuropathol Commun. 2023 Mar 30;11(1):53. doi: 10.1186/s40478-023-01551-8. Acta Neuropathol Commun. 2023. PMID: 36997967 Free PMC article.