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97 results

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Page 1
Quantitative Muscle Analysis in FSHD Using Whole-Body Fat-Referenced MRI: Composite Scores for Longitudinal and Cross-sectional Analysis.
Mellion ML, Widholm P, Karlsson M, Ahlgren A, Tawil R, Wagner KR, Statland JM, Wang L, Shieh PB, van Engelen BGM, Kools J, Ronco L, Odueyungbo A, Jiang J, Han JJ, Hatch M, Towles J, Leinhard OD, Cadavid D. Mellion ML, et al. Among authors: shieh pb. Neurology. 2022 Aug 30;99(9):e877-e889. doi: 10.1212/WNL.0000000000200757. Epub 2022 Jun 24. Neurology. 2022. PMID: 35750498
Randomized, placebo-controlled trials of dichlorphenamide in periodic paralysis.
Sansone VA, Burge J, McDermott MP, Smith PC, Herr B, Tawil R, Pandya S, Kissel J, Ciafaloni E, Shieh P, Ralph JW, Amato A, Cannon SC, Trivedi J, Barohn R, Crum B, Mitsumoto H, Pestronk A, Meola G, Conwit R, Hanna MG, Griggs RC; Muscle Study Group. Sansone VA, et al. Neurology. 2016 Apr 12;86(15):1408-1416. doi: 10.1212/WNL.0000000000002416. Epub 2016 Feb 10. Neurology. 2016. PMID: 26865514 Free PMC article. Clinical Trial.
Review of the Diagnosis and Treatment of Periodic Paralysis.
Statland JM, Fontaine B, Hanna MG, Johnson NE, Kissel JT, Sansone VA, Shieh PB, Tawil RN, Trivedi J, Cannon SC, Griggs RC. Statland JM, et al. Among authors: shieh pb. Muscle Nerve. 2018 Apr;57(4):522-530. doi: 10.1002/mus.26009. Epub 2017 Nov 29. Muscle Nerve. 2018. PMID: 29125635 Free PMC article. Review.
Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study.
LoRusso S, Johnson NE, McDermott MP, Eichinger K, Butterfield RJ, Carraro E, Higgs K, Lewis L, Mul K, Sacconi S, Sansone VA, Shieh P, van Engelen B, Wagner K, Wang L, Statland JM, Tawil R; ReSolve Investigators and the FSHD CTRN18. LoRusso S, et al. BMC Neurol. 2019 Sep 10;19(1):224. doi: 10.1186/s12883-019-1452-x. BMC Neurol. 2019. PMID: 31506080 Free PMC article.
The care of patients with Duchenne, Becker, and other muscular dystrophies in the COVID-19 pandemic.
Veerapandiyan A, Wagner KR, Apkon S, McDonald CM, Mathews KD, Parsons JA, Wong BL, Eichinger K, Shieh PB, Butterfield RJ, Rao VK, Smith EC, Proud CM, Connolly AM, Ciafaloni E. Veerapandiyan A, et al. Among authors: shieh pb. Muscle Nerve. 2020 Jul;62(1):41-45. doi: 10.1002/mus.26902. Epub 2020 May 5. Muscle Nerve. 2020. PMID: 32329920 Free PMC article. Review.
Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study.
Rudnicki SA, Andrews JA, Duong T, Cockroft BM, Malik FI, Meng L, Wei J, Wolff AA, Genge A, Johnson NE, Tesi-Rocha C, Connolly AM, Darras BT, Felice K, Finkel RS, Shieh PB, Mah JK, Statland J, Campbell C, Habib AA, Kuntz NL, Oskoui M, Day JW. Rudnicki SA, et al. Among authors: shieh pb. Neurotherapeutics. 2021 Apr;18(2):1127-1136. doi: 10.1007/s13311-020-01004-3. Epub 2021 Feb 23. Neurotherapeutics. 2021. PMID: 33624184 Free PMC article. Clinical Trial.
Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double-blind, placebo-controlled, dose-titration trial.
Wagner KR, Kuntz NL, Koenig E, East L, Upadhyay S, Han B, Shieh PB. Wagner KR, et al. Among authors: shieh pb. Muscle Nerve. 2021 Sep;64(3):285-292. doi: 10.1002/mus.27347. Epub 2021 Jun 29. Muscle Nerve. 2021. PMID: 34105177 Free PMC article. Clinical Trial.
Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial.
McDonald CM, Shieh PB, Abdel-Hamid HZ, Connolly AM, Ciafaloni E, Wagner KR, Goemans N, Mercuri E, Khan N, Koenig E, Malhotra J, Zhang W, Han B, Mendell JR; the Italian DMD Telethon Registry Study Group, Leuven NMRC Registry Investigators, CINRG Duchenne Natural History Investigators, and PROMOVI Trial Clinical Investigators. McDonald CM, et al. Among authors: shieh pb. J Neuromuscul Dis. 2021;8(6):989-1001. doi: 10.3233/JND-210643. J Neuromuscul Dis. 2021. PMID: 34120909 Free PMC article. Clinical Trial.
A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment.
Mitelman O, Abdel-Hamid HZ, Byrne BJ, Connolly AM, Heydemann P, Proud C, Shieh PB, Wagner KR, Dugar A, Santra S, Signorovitch J, Goemans N; investigators from the LNMRC Natural History study; McDonald CM; investigators from the CINRG Duchenne National History Study; Mercuri E; investigators from The DMD Italian Group; Mendell JR. Mitelman O, et al. Among authors: shieh pb. J Neuromuscul Dis. 2022;9(1):39-52. doi: 10.3233/JND-210665. J Neuromuscul Dis. 2022. PMID: 34420980 Free PMC article. Clinical Trial.
97 results