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Year | Number of Results |
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2017 | 2 |
2019 | 1 |
2020 | 1 |
2021 | 1 |
2024 | 0 |
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Short-duration splice promoting compound enables a tunable mouse model of spinal muscular atrophy.
Life Sci Alliance. 2020 Nov 24;4(1):e202000889. doi: 10.26508/lsa.202000889. Print 2021 Jan.
Life Sci Alliance. 2020.
PMID: 33234679
Free PMC article.
Optimization of a series of heterocycles as survival motor neuron gene transcription enhancers.
Choi S, Calder AN, Miller EH, Anderson KP, Fiejtek DK, Rietz A, Li H, Cherry JJ, Quist KM, Xing X, Glicksman MA, Cuny GD, Lorson CL, Androphy EA, Hodgetts KJ.
Choi S, et al.
Bioorg Med Chem Lett. 2017 Dec 1;27(23):5144-5148. doi: 10.1016/j.bmcl.2017.10.066. Epub 2017 Oct 26.
Bioorg Med Chem Lett. 2017.
PMID: 29103974
Free PMC article.
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Discovery of a Small Molecule Probe That Post-Translationally Stabilizes the Survival Motor Neuron Protein for the Treatment of Spinal Muscular Atrophy.
Rietz A, Li H, Quist KM, Cherry JJ, Lorson CL, Burnett BG, Kern NL, Calder AN, Fritsche M, Lusic H, Boaler PJ, Choi S, Xing X, Glicksman MA, Cuny GD, Androphy EJ, Hodgetts KJ.
Rietz A, et al.
J Med Chem. 2017 Jun 8;60(11):4594-4610. doi: 10.1021/acs.jmedchem.6b01885. Epub 2017 May 19.
J Med Chem. 2017.
PMID: 28481536
Free PMC article.
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Intraperitoneal delivery of a novel drug-like compound improves disease severity in severe and intermediate mouse models of Spinal Muscular Atrophy.
Osman EY, Rietz A, Kline RA, Cherry JJ, Hodgetts KJ, Lorson CL, Androphy EJ.
Osman EY, et al.
Sci Rep. 2019 Feb 7;9(1):1633. doi: 10.1038/s41598-018-38208-9.
Sci Rep. 2019.
PMID: 30733501
Free PMC article.
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