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Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy.
Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, Rodino-Klapac LR, Sahenk Z, Schnell FJ, Young GD, Mendell JR, Lowes LP. Alfano LN, et al. Among authors: frank de. Medicine (Baltimore). 2019 Jun;98(26):e15858. doi: 10.1097/MD.0000000000015858. Medicine (Baltimore). 2019. PMID: 31261494 Free PMC article. Clinical Trial.
Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy.
Frank DE, Schnell FJ, Akana C, El-Husayni SH, Desjardins CA, Morgan J, Charleston JS, Sardone V, Domingos J, Dickson G, Straub V, Guglieri M, Mercuri E, Servais L, Muntoni F; SKIP-NMD Study Group. Frank DE, et al. 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.
Validation of a Muscle-Specific Tissue Image Analysis Tool for Quantitative Assessment of Dystrophin Staining in Frozen Muscle Biopsies.
Aeffner F, Faelan C, Moore SA, Moody A, Black JC, Charleston JS, Frank DE, Dworzak J, Piper JK, Ranjitkar M, Wilson K, Kanaly S, Rudmann DG, Lange H, Young GD, Milici AJ. Aeffner F, et al. Among authors: frank de. Arch Pathol Lab Med. 2019 Feb;143(2):197-205. doi: 10.5858/arpa.2017-0536-OA. Epub 2018 Aug 31. Arch Pathol Lab Med. 2019. PMID: 30168727 Free PMC article.
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. Among authors: frank de. 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.
Mild mitochondrial metabolic deficits by α-ketoglutarate dehydrogenase inhibition cause prominent changes in intracellular autophagic signaling: Potential role in the pathobiology of Alzheimer's disease.
Banerjee K, Munshi S, Xu H, Frank DE, Chen HL, Chu CT, Yang J, Cho S, Kagan VE, Denton TT, Tyurina YY, Jiang JF, Gibson GE. Banerjee K, et al. Among authors: frank de. Neurochem Int. 2016 Jun;96:32-45. doi: 10.1016/j.neuint.2016.02.011. Epub 2016 Feb 23. Neurochem Int. 2016. PMID: 26923918 Free PMC article.
62 results