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Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy.
Neurology. 2020 May 26;94(21):e2270-e2282. doi: 10.1212/WNL.0000000000009233. Epub 2020 Mar 5.
Neurology. 2020.
PMID: 32139505
Free PMC article.
Clinical Trial.
Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial.
Servais L, Mercuri E, Straub V, Guglieri M, Seferian AM, Scoto M, Leone D, Koenig E, Khan N, Dugar A, Wang X, Han B, Wang D, Muntoni F; SKIP-NMD Study Group.
Servais L, et al.
Nucleic Acid Ther. 2022 Feb;32(1):29-39. doi: 10.1089/nat.2021.0043. Epub 2021 Nov 17.
Nucleic Acid Ther. 2022.
PMID: 34788571
Free PMC article.
Clinical Trial.
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Low-level dystrophin expression attenuating the dystrophinopathy phenotype.
Waldrop MA, Gumienny F, El Husayni S, Frank DE, Weiss RB, Flanigan KM.
Waldrop MA, et al.
Neuromuscul Disord. 2018 Feb;28(2):116-121. doi: 10.1016/j.nmd.2017.11.007. Epub 2017 Nov 23.
Neuromuscul Disord. 2018.
PMID: 29305136
Free PMC article.
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CCL5 derived from platelets increases megakaryocyte proplatelet formation.
Machlus KR, Johnson KE, Kulenthirarajan R, Forward JA, Tippy MD, Soussou TS, El-Husayni SH, Wu SK, Wang S, Watnick RS, Italiano JE Jr, Battinelli EM.
Machlus KR, et al. Among authors: el husayni sh.
Blood. 2016 Feb 18;127(7):921-6. doi: 10.1182/blood-2015-05-644583. Epub 2015 Dec 8.
Blood. 2016.
PMID: 26647394
Free PMC article.
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