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Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study.
van der Ploeg A, Carlier PG, Carlier RY, Kissel JT, Schoser B, Wenninger S, Pestronk A, Barohn RJ, Dimachkie MM, Goker-Alpan O, Mozaffar T, Pena LD, Simmons Z, Straub V, Guglieri M, Young P, Boentert M, Baudin PY, Wens S, Shafi R, Bjartmar C, Thurberg BL. van der Ploeg A, et al. Among authors: shafi r. Mol Genet Metab. 2016 Sep;119(1-2):115-23. doi: 10.1016/j.ymgme.2016.05.013. Epub 2016 May 19. Mol Genet Metab. 2016. PMID: 27473031 Free article. Clinical Trial.
Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study.
Pena LDM, Barohn RJ, Byrne BJ, Desnuelle C, Goker-Alpan O, Ladha S, Laforêt P, Mengel KE, Pestronk A, Pouget J, Schoser B, Straub V, Trivedi J, Van Damme P, Vissing J, Young P, Kacena K, Shafi R, Thurberg BL, Culm-Merdek K, van der Ploeg AT; NEO1 Investigator Group. Pena LDM, et al. Among authors: shafi r. Neuromuscul Disord. 2019 Mar;29(3):167-186. doi: 10.1016/j.nmd.2018.12.004. Epub 2018 Dec 17. Neuromuscul Disord. 2019. PMID: 30770310 Free article. Clinical Trial.
Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study.
Hahn SH, Kronn D, Leslie ND, Pena LDM, Tanpaiboon P, Gambello MJ, Gibson JB, Hillman R, Stockton DW, Day JW, Wang RY, An Haack K, Shafi R, Sparks S, Zhao Y, Wilson C, Kishnani PS; Pompe ADVANCE Study Consortium. Hahn SH, et al. Among authors: shafi r. Genet Med. 2018 Oct;20(10):1284-1294. doi: 10.1038/gim.2018.2. Epub 2018 Mar 22. Genet Med. 2018. PMID: 29565424 Free article. Clinical Trial.
Social Media and Youth Mental Health.
Weigle PE, Shafi RMA. Weigle PE, et al. Among authors: shafi rma. Curr Psychiatry Rep. 2024 Jan;26(1):1-8. doi: 10.1007/s11920-023-01478-w. Epub 2023 Dec 16. Curr Psychiatry Rep. 2024. PMID: 38103128 Review.
The Importance of Sibling Relationships for Children in Foster Care.
Shafi RMA, Pease ER, Romanowicz M, Baker M, Wakefield S, Morgan WS, Dell ML. Shafi RMA, et al. J Am Acad Psychiatry Law. 2023 Jun;51(2):255-262. doi: 10.29158/JAAPL.230029-23. Epub 2023 May 31. J Am Acad Psychiatry Law. 2023. PMID: 37257906
Early Detection of Forest Fire Using Mixed Learning Techniques and UAV.
Kasyap VL, Sumathi D, Alluri K, Reddy Ch P, Thilakarathne N, Shafi RM. Kasyap VL, et al. Among authors: shafi rm. Comput Intell Neurosci. 2022 Jul 9;2022:3170244. doi: 10.1155/2022/3170244. eCollection 2022. Comput Intell Neurosci. 2022. PMID: 35855796 Free PMC article.
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