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Implications of circulating neurofilaments for spinal muscular atrophy treatment early in life: A case series.
Alves CRR, Petrillo M, Spellman R, Garner R, Zhang R, Kiefer M, Simeone S, Sohn J, Eichelberger EJ, Rodrigues E, Arruda EA, Townsend EL, Farwell W, Swoboda KJ. Alves CRR, et al. Among authors: kiefer m. Mol Ther Methods Clin Dev. 2021 Oct 30;23:524-538. doi: 10.1016/j.omtm.2021.10.011. eCollection 2021 Dec 10. Mol Ther Methods Clin Dev. 2021. PMID: 34853799 Free PMC article.
Development of Duchenne Video Assessment scorecards to evaluate ease of movement among those with Duchenne muscular dystrophy.
Contesse MG, Lowes LP, White MK, Dalle Pazze L, McSherry C, Alfano LN, Iammarino M, Reash N, Bonarrigo K, Kiefer M, Laubscher K, McIntyre M, Mockler S, Nelson L, Vogel L, Leffler MG. Contesse MG, et al. Among authors: kiefer m. PLoS One. 2022 Apr 13;17(4):e0266845. doi: 10.1371/journal.pone.0266845. eCollection 2022. PLoS One. 2022. PMID: 35417501 Free PMC article.
RNA mis-splicing in children with congenital myotonic dystrophy is associated with physical function.
Hartman JM, Ikegami K, Provenzano M, Bates K, Butler A, Jones AS, Berggren KN, Dekdebrun J, McKay MJ, Baldwin JN, Cornett KMD, Burns J, Kiefer M, Johnson NE, Hale MA; DMCRN Consortium. Hartman JM, et al. Among authors: kiefer m. Ann Clin Transl Neurol. 2024 Dec;11(12):3175-3191. doi: 10.1002/acn3.52224. Epub 2024 Oct 25. Ann Clin Transl Neurol. 2024. PMID: 39450929 Free PMC article.
433 results