Tawil R, Wagner KR, Hamel JI, Leung DG, Statland JM, Wang LH, Genge A, Sacconi S, Lochmüller H, Reyes-Leiva D, Diaz-Manera J, Alonso-Perez J, Muelas N, Vilchez JJ, Pestronk A, Gibson S, Goyal NA, Hayward LJ, Johnson N, LoRusso S, Freimer M, Shieh PB, Subramony SH, van Engelen B, Kools J, Leinhard OD, Widholm P, Morabito C, Moxham CM, Cadavid D, Mellion ML, Odueyungbo A, Tracewell WG, Accorsi A, Ronco L, Gould RJ, Shoskes J, Rojas LA, Jiang JG. Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trial. Lancet Neurol. 2024 May;23(5):477-486. doi: 10.1016/S1474-4422(24)00073-5. PubMed PMID: 38631764.

Wang LH, Leung DG, Wagner KR, Lowry SJ, McDermott MP, Eichinger K, Higgs K, Walker M, Lewis L, Martens WB, Mul K, Sansone VA, Shieh P, Elsheikh B, LoRusso S, Butterfield RJ, Johnson N, Preston MR, Messina C, Carraro E, Tawil R, Statland J. Lean tissue mass measurements by dual-energy X-ray absorptiometry and associations with strength and functional outcome measures in facioscapulohumeral muscular dystrophy. Neuromuscul Disord. 2023 Sep;33(9):63-68. doi: 10.1016/j.nmd.2023.06.008. Epub 2023 Jun 21. PubMed PMID: 37400350; PubMed Central PMCID: PMC10527411.

Mohassel P, Yun P, Syeda S, Batra A, Bradley AJ, Donkervoort S, Monges S, Cohen JS, Leung DG, Munell F, Ortez C, Sánchez-Montáñez A, Karachunski P, Brandsema J, Medne L, Chaudhry V, Tasca G, Foley AR, Udd B, Arai AE, Walter GA, Bönnemann CG. A comprehensive study of skeletal muscle imaging in FHL1-related reducing body myopathy. Ann Clin Transl Neurol. 2023 Aug;10(8):1442-1455. doi: 10.1002/acn3.51834. Epub 2023 Jul 22. PubMed PMID: 37483011; PubMed Central PMCID: PMC10424657.

Thornton CA, Moxley RT 3rd, Eichinger K, Heatwole C, Mignon L, Arnold WD, Ashizawa T, Day JW, Dent G, Tanner MK, Duong T, Greene EP, Herbelin L, Johnson NE, King W, Kissel JT, Leung DG, Lott DJ, Norris DA, Pucillo EM, Schell W, Statland JM, Stinson N, Subramony SH, Xia S, Bishop KM, Bennett CF. Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial. Lancet Neurol. 2023 Mar;22(3):218-228. doi: 10.1016/S1474-4422(23)00001-7. PubMed PMID: 36804094.

Leung DG, Kinoshita J, Arjomand J, Dumonceaux J. Meeting report: The 2022 FSHD International Research Congress. Neuromuscul Disord. 2023 Feb;33(2):196-198. doi: 10.1016/j.nmd.2022.12.005. Epub 2022 Dec 13. PubMed PMID: 36682112.

Amanat M, Nemeth CL, Fine AS, Leung DG, Fatemi A. Antisense Oligonucleotide Therapy for the Nervous System: From Bench to Bedside with Emphasis on Pediatric Neurology. Pharmaceutics. 2022 Nov 5;14(11). doi: 10.3390/pharmaceutics14112389. Review. PubMed PMID: 36365206; PubMed Central PMCID: PMC9695718.

Leung DG, Bocchieri AE, Ahlawat S, Jacobs MA, Parekh VS, Braverman V, Summerton K, Mansour J, Stinson N, Bibat G, Morris C, Marraffino S, Wagner KR. A phase Ib/IIa, open-label, multiple ascending-dose trial of domagrozumab in fukutin-related protein limb-girdle muscular dystrophy. Muscle Nerve. 2021 Aug;64(2):172-179. doi: 10.1002/mus.27259. Epub 2021 May 24. PubMed PMID: 33961310.

Leung DG, Bocchieri AE, Ahlawat S, Jacobs MA, Parekh VS, Braverman V, Summerton K, Mansour J, Bibat G, Morris C, Marraffino S, Wagner KR. Longitudinal functional and imaging outcome measures in FKRP limb-girdle muscular dystrophy. BMC Neurol. 2020 May 19;20(1):196. doi: 10.1186/s12883-020-01774-5. PubMed PMID: 32429923; PubMed Central PMCID: PMC7236878.

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. Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study. BMC Neurol. 2019 Sep 10;19(1):224. doi: 10.1186/s12883-019-1452-x. PubMed PMID: 31506080; PubMed Central PMCID: PMC6734593.

Leung DG, Wang X, Barker PB, Carrino JA, Wagner KR. Multivoxel proton magnetic resonance spectroscopy in facioscapulohumeral muscular dystrophy. Muscle Nerve. 2018 Jun;57(6):958-963. doi: 10.1002/mus.26048. Epub 2018 Feb 14. PubMed PMID: 29266323; PubMed Central PMCID: PMC5951728.

Leung DG. Magnetic resonance imaging in facioscapulohumeral muscular dystrophy. Muscle Nerve. 2018 Jun;57(6):872-874. doi: 10.1002/mus.26064. Epub 2018 Jan 23. PubMed PMID: 29328515; PubMed Central PMCID: PMC5951734.

Kalia V, Leung DG, Sneag DB, Del Grande F, Carrino JA. Advanced MRI Techniques for Muscle Imaging. Semin Musculoskelet Radiol. 2017 Sep;21(4):459-469. doi: 10.1055/s-0037-1604007. Epub 2017 Aug 3. Review. PubMed PMID: 28772322; PubMed Central PMCID: PMC5749928.

Leung DG. Magnetic resonance imaging patterns of muscle involvement in genetic muscle diseases: a systematic review. J Neurol. 2017 Jul;264(7):1320-1333. doi: 10.1007/s00415-016-8350-6. Epub 2016 Nov 25. Review. PubMed PMID: 27888415; PubMed Central PMCID: PMC5445016.

Wong EG, Parker AM, Leung DG, Brigham EP, Arbaje AI. Association of severity of illness and intensive care unit readmission: A systematic review. Heart Lung. 2016 Jan-Feb;45(1):3-9.e2. doi: 10.1016/j.hrtlng.2015.10.040. Review. PubMed PMID: 26702501; PubMed Central PMCID: PMC4692266.

Leung DG, Carrino JA, Wagner KR, Jacobs MA. Whole-body magnetic resonance imaging evaluation of facioscapulohumeral muscular dystrophy. Muscle Nerve. 2015 Oct;52(4):512-20. doi: 10.1002/mus.24569. Epub 2015 Mar 31. PubMed PMID: 25641525; PubMed Central PMCID: PMC4504833.

Faridian-Aragh N, Wagner KR, Leung DG, Carrino JA. Magnetic resonance imaging phenotyping of Becker muscular dystrophy. Muscle Nerve. 2014 Dec;50(6):962-7. doi: 10.1002/mus.24246. Epub 2014 Oct 30. PubMed PMID: 24659522.

Leung DG, Herzka DA, Thompson WR, He B, Bibat G, Tennekoon G, Russell SD, Schuleri KH, Lardo AC, Kass DA, Thompson RE, Judge DP, Wagner KR. Sildenafil does not improve cardiomyopathy in Duchenne/Becker muscular dystrophy. Ann Neurol. 2014 Oct;76(4):541-9. doi: 10.1002/ana.24214. Epub

Leung DG, Taylor HA, Lindy AS, Basehore MJ, Mammen AL. A case of progressive quadriceps weakness and elevated creatine kinase level mimicking inclusion body myositis. Arthritis Care Res (Hoboken). 2014 Feb;66(2):328-33. doi: 10.1002/acr.22156. PubMed PMID: 24022920.

Leung DG, Wagner KR. Therapeutic advances in muscular dystrophy. Ann Neurol. 2013 Sep;74(3):404-11. doi: 10.1002/ana.23989. Review. PubMed PMID: 23939629; PubMed Central PMCID: PMC3886293.

Rahimov F, King OD, Leung DG, Bibat GM, Emerson CP Jr, Kunkel LM, Wagner KR. Transcriptional profiling in facioscapulohumeral muscular dystrophy to identify candidate biomarkers. Proc Natl Acad Sci U S A. 2012 Oct 2;109(40):16234-9. doi: 10.1073/pnas.1209508109. Epub 2012 Sep 17. PubMed PMID: 22988124; PubMed Central PMCID: PMC3479603.

Gilbert-Honick J, Ginn B, Zhang Y, Salehi S, Wagner KR, Mao HQ, Grayson WL.  Adipose-derived stem/stromal cells on electrospun fibrin microfiber bundles enable moderate muscle reconstruction in a volumetric muscle loss model.  Cell Transplant.  2018 Oct 9:  963689718805370.  PMID:  30298751.

Estrellas KM, Chung L, Cheu LA, Sadtler K, Majumdar S, Mula J, Wolf MT, Elisseeff JH, Wagner KR.  Biological scaffold-mediated delivery of myostatin inhibitor promotes a regenerative immune response in an animal model of Duchenne muscular dystrophy.  J Biol Chem.  2018 Oct 5, 293 (4): 15594-15605.  PMID 30139748. 

Chen JCJ, King OD, Zhang Y, Clayton NP, Spencer C, Wentworth BM, Emerson CP, Wagner KR. Morpholino-Mediated Knockdown of DUX4 Towards Facioscapulohumeral Muscular Dystrophy Therapeutics.  Molecular Therapy 2016, 24(8):1405-11.  PMID 27378237.

Choudhury SR, Fitzpatrick Z, Harris, AF, Maitland SA, Ferreira JS, Zhang Y, Ma S, Sharma RB, Ray-Edwards HL, Johnson JA, Johnson AK, Alonso LC, Punzo, C, Wagner KR, Maguire CA, Kotin RM, Martin DR, Sena-Esteves M.  In vivo selection yields AAV-B1 capsids for CNS and muscle gene therapy.  Mol Ther. 2016, 24 (7): 1247-57. PMID 27117222.

Choi IY, Lim H, Estrellas K, Mula J, Cohen T, Zhang Y, Donnelly C, Richard JP, Kin Y, Kim H, Kazuki Y, Oshimura M, Rothstein J, Maragakis N, Wagner KR, Lee G. Concordant but varied phenotypes among patient-specific myoblasts of Duchenne muscular dystrophy revealed by human iPSC-based model.  Cell Reports  2016, 15(1): 2301-12. PMID 27239027. 

Kornegay JN, Bogan DJ, Bogan JR, Dow JL, Wang J, Fan Z, Liu N, Warsing L, Grange RW, Ahn M, Balog-Alvarez J, Cotton SW, Willis MS, Brinkmeyer-Langford C, Zhu H, Palandra J, Morris CA, Styner MA, Wagner KR.  Dystrophin-deficient dogs with reduced myostatin have unequal muscle growth and greater joint contractures.  Skeletal Muscle  2016, 6:14.  PMID:27047655 

Moyer AL and Wagner KR.  Mammalian Mss51 is a skeletal muscle-specific gene modulating cellular metabolism.  J Neuromusc Disease.  2015, 2(4):  371-385. PMID26634192.  

Zhang Y, King OD, Rahimov, F, Jones TI, Ward CW, Kerr JP, Liu N, Emerson CP Jr, Kunkel LM, Partridge TA, Wagner KR.  Human skeletal muscle xenograft as a new preclinical model for muscle disorders.  Hum Mol Genet. 2014, 23 (12): 3180-3188.  PMID 24452336.

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