1.
Morren JA, Galvez-Jimenez N. Current and prospective disease-modifying therapies for amyotrophic lateral sclerosis. Expert Opinion on Investigational Drugs. 2012 Mar;21(3):297–320.
2.
Mitsumoto H, Brooks BR, Silani V. Clinical trials in amyotrophic lateral sclerosis: why so many negative trials and how can trials be improved? The Lancet Neurology. 2014 Nov;13(11):1127–1138.
3.
Maragakis NJ. What can we learn from the edaravone development program for ALS? Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 2017 Oct 31;18(sup1):98–103.
4.
Hughes J, Rees S, Kalindjian S, Philpott K. Principles of early drug discovery. British Journal of Pharmacology. 2011 Mar;162(6):1239–1249.
5.
Birnkrant DJ, Bushby K, Bann CM, Apkon SD, Blackwell A, Brumbaugh D, Case LE, Clemens PR, Hadjiyannakis S, Pandya S, Street N, Tomezsko J, Wagner KR, Ward LM, Weber DR. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. The Lancet Neurology. 2018 Mar;17(3):251–267.
6.
Birnkrant DJ, Bushby K, Bann CM, Alman BA, Apkon SD, Blackwell A, Case LE, Cripe L, Hadjiyannakis S, Olson AK, Sheehan DW, Bolen J, Weber DR, Ward LM. Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management. The Lancet Neurology. 2018 Apr;17(4):347–361.
7.
Birnkrant DJ, Bushby K, Bann CM, Apkon SD, Blackwell A, Colvin MK, Cripe L, Herron AR, Kennedy A, Kinnett K, Naprawa J, Noritz G, Poysky J, Street N, Trout CJ, Weber DR, Ward LM. Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across the lifespan. The Lancet Neurology. 2018 May;17(5):445–455.
8.
Mercuri E, Finkel RS, Muntoni F, Wirth B, Montes J, Main M, Mazzone ES, Vitale M, Snyder B, Quijano-Roy S, Bertini E, Davis RH, Meyer OH, Simonds AK, Schroth MK, Graham RJ, Kirschner J, Iannaccone ST, Crawford TO, Woods S, Qian Y, Sejersen T, Muntoni F, Wirth B, Tiziano FD, Kirschner J, Tizzano E, Topaloglu H, Swoboda K, Laing N, Kayoko S, Prior T, Chung WK, Wu SM, Montes J, Mazzone E, Main M, Coleman C, Gee R, Glanzman A, Kroksmark AK, Krosschell K, Nelson L, Rose K, Stępień A, Vuillerot C, Vitale M, Snyder B, Quijano-Roy S, Dubousset J. Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care. Neuromuscular Disorders. 2018 Feb;28(2):103–115.
9.
Finkel RS, Mercuri E, Meyer OH, Simonds AK, Schroth MK, Graham RJ, Kirschner J, Iannaccone ST, Crawford TO, Woods S, Muntoni F, Wirth B, Montes J, Main M, Mazzone ES, Vitale M, Snyder B, Quijano-Roy S, Bertini E, Davis RH, Qian Y, Sejersen T. Diagnosis and management of spinal muscular atrophy: Part 2: Pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics. Neuromuscular Disorders. 2018 Mar;28(3):197–207.
10.
Scoto M, Finkel RS, Mercuri E, Muntoni F. Therapeutic approaches for spinal muscular atrophy (SMA). Gene Therapy. 2017 Sep;24(9):514–519.
11.
Ramsey D, Scoto M, Mayhew A, Main M, Mazzone ES, Montes J, de Sanctis R, Dunaway Young S, Salazar R, Glanzman AM, Pasternak A, Quigley J, Mirek E, Duong T, Gee R, Civitello M, Tennekoon G, Pane M, Pera MC, Bushby K, Day J, Darras BT, De Vivo D, Finkel R, Mercuri E, Muntoni F. Revised Hammersmith Scale for spinal muscular atrophy: A SMA specific clinical outcome assessment tool. PLOS ONE. 2017 Feb 21;12(2).
12.
Mazzone ES, Mayhew A, Montes J, Ramsey D, Fanelli L, Young SD, Salazar R, De Sanctis R, Pasternak A, Glanzman A, Coratti G, Civitello M, Forcina N, Gee R, Duong T, Pane M, Scoto M, Pera MC, Messina S, Tennekoon G, Day JW, Darras BT, De Vivo DC, Finkel R, Muntoni F, Mercuri E. Revised upper limb module for spinal muscular atrophy: Development of a new module. Muscle & Nerve. 2017 Jun;55(6):869–874.
13.
Westerberg E, Molin CJ, Spörndly Nees S, Widenfalk J, Punga AR. The impact of physical exercise on neuromuscular function in Myasthenia gravis patients. Medicine. 2018 Aug;97(31).
14.
Peragallo JH. Pediatric Myasthenia Gravis. Seminars in Pediatric Neurology. 2017 May;24(2):116–121.
15.
Laurá M, Singh D, Ramdharry G, Morrow J, Skorupinska M, Pareyson D, Burns J, Lewis RA, Scherer SS, Herrmann DN, Cullen N, Bradish C, Gaiani L, Martinelli N, Gibbons P, Pfeffer G, Phisitkul P, Wapner K, Sanders J, Flemister S, Shy ME, Reilly MM. Prevalence and orthopedic management of foot and ankle deformities in Charcot-Marie-Tooth disease. Muscle & Nerve. 2018 Feb;57(2):255–259.
16.
Reilly MM, Pareyson D, Burns J, Laurá M, Shy ME, Singh D, Agren PH, Altmann V, Baets J, Briggs P, Burns J, Butcher K, Gaiani L, Genovese F, Gibbons P, Laurá M, Louwerens JW, Manzur A, Moroni I, Martinelli N, Pareyson D, Pfeffer G, Ramdharry G, Reilly MM, Shy M, Singh D, van der Linden M, Wenz W. 221st ENMC International Workshop: Neuromuscular Disorders. 2017 Dec;27(12):1138–1142.
17.
Ramdharry GM, Pollard A, Anderson C, Laurá M, Murphy SM, Dudziec M, Dewar EL, Hutton E, Grant R, Reilly MM. A pilot study of proximal strength training in Charcot-Marie-Tooth disease. Journal of the Peripheral Nervous System. 2014 Dec;19(4):328–332.
18.
Gibson S, Haringer V. Amyotrophic lateral sclerosis: clinical perspectives. Orphan Drugs: Research and Reviews. 2015 Apr;
19.
Berlowitz DJ, Howard ME, Fiore JF, Vander Hoorn S, O’Donoghue FJ, Westlake J, Smith A, Beer F, Mathers S, Talman P. Identifying who will benefit from non-invasive ventilation in amyotrophic lateral sclerosis/motor neurone disease in a clinical cohort. Journal of Neurology, Neurosurgery & Psychiatry. 2016 Mar;87(3):280–286.
20.
Harwood CA, McDermott CJ, Shaw PJ. Clinical aspects of motor neurone disease. Medicine. 2012 Oct;40(10):540–545.
21.
Drory VE, Goltsman E, Goldman Reznik J, Mosek A, Korczyn AD. The value of muscle exercise in patients with amyotrophic lateral sclerosis. Journal of the Neurological Sciences. 2001 Oct;191(1–2):133–137.
22.
Al-Chalabi A, van den Berg LH, Veldink J. Gene discovery in amyotrophic lateral sclerosis: implications for clinical management. Nature Reviews Neurology. 2017 Feb;13(2):96–104.
23.
Carrì MT, D’Ambrosi N, Cozzolino M. Pathways to mitochondrial dysfunction in ALS pathogenesis. Biochemical and Biophysical Research Communications. 2017 Feb;483(4):1187–1193.
24.
Lin G, Mao D, Bellen HJ. Amyotrophic Lateral Sclerosis Pathogenesis Converges on Defects in Protein Homeostasis Associated with TDP-43 Mislocalization and Proteasome-Mediated Degradation Overload. Fly Models of Human Diseases [Internet]. Elsevier; 2017. p. 111–171. Available from: https://linkinghub.elsevier.com/retrieve/pii/S0070215316301454
25.
Monahan Z, Shewmaker F, Pandey UB. Stress granules at the intersection of autophagy and ALS. Brain Research. 2016 Oct;1649:189–200.
26.
Ruegsegger C, Saxena S. Proteostasis impairment in ALS. Brain Research. 2016 Oct;1648:571–579.
27.
Renton AE, Chiò A, Traynor BJ. State of play in amyotrophic lateral sclerosis genetics. Nature Neuroscience. 2014 Jan;17(1):17–23.
28.
Jessell TM. Neuronal specification in the spinal cord: inductive signals and transcriptional codes. Nature Reviews Genetics. 2000 Oct;1(1):20–29.
29.
Harland R. Neural induction. Current Opinion in Genetics & Development. 2000 Aug;10(4):357–362.
30.
Dasen JS, Jessell TM. Chapter Six Hox Networks and the Origins of Motor Neuron Diversity. Hox Genes [Internet]. Elsevier; 2009. p. 169–200. Available from: https://linkinghub.elsevier.com/retrieve/pii/S007021530988006X
31.
Bonanomi D, Pfaff SL. Motor Axon Pathfinding. Cold Spring Harbor Perspectives in Biology. 2010 Mar 1;2(3):a001735–a001735.
32.
Darabid H, Perez-Gonzalez AP, Robitaille R. Neuromuscular synaptogenesis: coordinating partners with multiple functions. Nature Reviews Neuroscience. 2014 Nov;15(11):703–718.
33.
Kanning KC, Kaplan A, Henderson CE. Motor Neuron Diversity in Development and Disease. Annual Review of Neuroscience. 2010 Jun;33(1):409–440.
34.
Ladle DR, Pecho-Vrieseling E, Arber S. Assembly of Motor Circuits in the Spinal Cord: Driven to Function by Genetic and Experience-Dependent Mechanisms. Neuron. 2007 Oct;56(2):270–283.
35.
Brownstone RM, Bui TV. Spinal interneurons providing input to the final common path during locomotion. Breathe, Walk and Chew: The Neural Challenge: Part I [Internet]. Elsevier; 2010. p. 81–95. Available from: https://linkinghub.elsevier.com/retrieve/pii/B978044453613600006X
36.
Li L, Xiong WC, Mei L. Neuromuscular Junction Formation, Aging, and Disorders. Annual Review of Physiology. 2018 Feb 10;80(1):159–188.
37.
Singhal N, Martin PT. Role of extracellular matrix proteins and their receptors in the development of the vertebrate neuromuscular junction. Developmental Neurobiology. 2011 Nov;71(11):982–1005.
38.
Nishimune H, Valdez G, Jarad G, Moulson CL, Müller U, Miner JH, Sanes JR. Laminins promote postsynaptic maturation by an autocrine mechanism at the neuromuscular junction. The Journal of Cell Biology. 2008 Sep 22;182(6):1201–1215.
39.
Rudolf R, Khan MM, Labeit S, Deschenes MR. Degeneration of Neuromuscular Junction in Age and Dystrophy. Frontiers in Aging Neuroscience. 2014 May 22;6.
40.
Jones RA, Harrison C, Eaton SL, Llavero Hurtado M, Graham LC, Alkhammash L, Oladiran OA, Gale A, Lamont DJ, Simpson H, Simmen MW, Soeller C, Wishart TM, Gillingwater TH. Cellular and Molecular Anatomy of the Human Neuromuscular Junction. Cell Reports. 2017 Nov;21(9):2348–2356.
41.
O’Connor E, Töpf A, Zahedi RP, Spendiff S, Cox D, Roos A, Lochmüller H. Clinical and research strategies for limb-girdle congenital myasthenic syndromes. Annals of the New York Academy of Sciences. 2018 Jan;1412(1):102–112.
42.
Engel AG, Shen XM, Selcen D, Sine SM. Congenital myasthenic syndromes: pathogenesis, diagnosis, and treatment. The Lancet Neurology. 2015 Apr;14(4):420–434.
43.
Cruz PMR, Palace J, Beeson D. Congenital myasthenic syndromes and the neuromuscular junction. Current Opinion in Neurology. 2014 Oct;27(5):566–575.
44.
Rodríguez Cruz PM, Palace J, Beeson D. Inherited disorders of the neuromuscular junction: an update. Journal of Neurology. 2014 Nov;261(11):2234–2243.
45.
Belaya K, Rodríguez Cruz PM, Liu WW, Maxwell S, McGowan S, Farrugia ME, Petty R, Walls TJ, Sedghi M, Basiri K, Yue WW, Sarkozy A, Bertoli M, Pitt M, Kennett R, Schaefer A, Bushby K, Parton M, Lochmüller H, Palace J, Muntoni F, Beeson D. Mutations in cause congenital myasthenic syndrome and bridge myasthenic disorders with dystroglycanopathies. Brain. 2015 Sep;138(9):2493–2504.
46.
Rodríguez Cruz PM, Sewry C, Beeson D, Jayawant S, Squier W, McWilliam R, Palace J. Congenital myopathies with secondary neuromuscular transmission defects; A case report and review of the literature. Neuromuscular Disorders. 2014 Dec;24(12):1103–1110.
47.
Crisp SJ, Kullmann DM, Vincent A. Autoimmune synaptopathies. Nature Reviews Neuroscience. 2016 Feb;17(2):103–117.
48.
Gilhus NE. Myasthenia Gravis. New England Journal of Medicine. 2016 Dec 29;375(26):2570–2581.
49.
Kusner LL, Kaminski HJ. Myasthenia Gravis. Neurobiology of Brain Disorders [Internet]. Elsevier; 2015. p. 135–150. Available from: https://linkinghub.elsevier.com/retrieve/pii/B9780123982704000100
50.
Leung DG. Other Proven and Putative Autoimmune Disorders of the Peripheral Nervous System [Internet]. Oxford University Press; 2017. Available from: http://oxfordmedicine.com/view/10.1093/med/9780199937837.001.0001/med-9780199937837-chapter-98
51.
Spillane J, Beeson DJ, Kullmann DM. Myasthenia and related disorders of the neuromuscular junction. Journal of Neurology, Neurosurgery & Psychiatry. 2010 Aug 1;81(8):850–857.
52.
Meriggioli MN, Sanders DB. Autoimmune myasthenia gravis: emerging clinical and biological heterogeneity. The Lancet Neurology. 2009 May;8(5):475–490.
53.
Spillane J, Ermolyuk Y, Cano-Jaimez M, Lang B, Vincent A, Volynski KE, Kullmann DM. Lambert-Eaton syndrome IgG inhibits transmitter release via P/Q Ca2+ channels. Neurology. 2015 Feb 10;84(6):575–579.
54.
Wolfe GI, Kaminski HJ, Aban IB, Minisman G, Kuo HC, Marx A, Ströbel P, Mazia C, Oger J, Cea JG, Heckmann JM, Evoli A, Nix W, Ciafaloni E, Antonini G, Witoonpanich R, King JO, Beydoun SR, Chalk CH, Barboi AC, Amato AA, Shaibani AI, Katirji B, Lecky BRF, Buckley C, Vincent A, Dias-Tosta E, Yoshikawa H, Waddington-Cruz M, Pulley MT, Rivner MH, Kostera-Pruszczyk A, Pascuzzi RM, Jackson CE, Garcia Ramos GS, Verschuuren JJGM, Massey JM, Kissel JT, Werneck LC, Benatar M, Barohn RJ, Tandan R, Mozaffar T, Conwit R, Odenkirchen J, Sonett JR, Jaretzki A, Newsom-Davis J, Cutter GR. Randomized Trial of Thymectomy in Myasthenia Gravis. New England Journal of Medicine. 2016 Aug 11;375(6):511–522.
55.
Orrell, Richard WBarclay, Chris. Diagnosis and management of motor neurone disease. Practitioner [Internet]. 260:17–21. Available from: https://search.proquest.com/docview/1844334383/64C39DCAF3D346C0PQ/1?accountid=14511
56.
Morgan S, Orrell RW. Pathogenesis of amyotrophic lateral sclerosis. British Medical Bulletin. 2016 Sep;119(1):87–98.
57.
Fuller G, Manford M. Neurology: an illustrated colour text [Internet]. 3rd ed. Edinburgh: Churchill Livingstone Elsevier; 2010. Available from: http://ucl.alma.exlibrisgroup.com/view/action/uresolver.do?operation=resolveService&package_service_id=3669595080004761&institutionId=4761&customerId=4760
58.
Motor neurone disease: assessment and management | Guidance and guidelines | NICE. NICE; Available from: https://www.nice.org.uk/guidance/ng42
59.
Couratier P, Corcia P, Lautrette G, Nicol M, Preux PM, Marin B. Epidemiology of amyotrophic lateral sclerosis: A review of literature. Revue Neurologique. 2016 Jan;172(1):37–45.
60.
Otto M, Bowser R, Turner M, Berry J, Brettschneider J, Connor J, Costa J, Cudkowicz M, Glass J, Jahn O, Lehnert S, Malaspina A, Parnetti L, Petzold A, Shaw P, Sherman A, Steinacker P, Süßmuth S, Teunissen C, Tumani H, Wuolikainen A, Ludolph A. Roadmap and standard operating procedures for biobanking and discovery of neurochemical markers in ALS. Amyotrophic Lateral Sclerosis. 2012 Jan;13(1):1–10.
61.
Lu CH, Macdonald-Wallis C, Gray E, Pearce N, Petzold A, Norgren N, Giovannoni G, Fratta P, Sidle K, Fish M, Orrell R, Howard R, Talbot K, Greensmith L, Kuhle J, Turner MR, Malaspina A. Neurofilament light chain: A prognostic biomarker in amyotrophic lateral sclerosis. Neurology. 2015 Jun 2;84(22):2247–2257.
62.
Benatar M, Boylan K, Jeromin A, Rutkove SB, Berry J, Atassi N, Bruijn L. ALS biomarkers for therapy development: State of the field and future directions. Muscle & Nerve. 2016 Feb;53(2):169–182.
63.
Andreasson U, Blennow K, Zetterberg H. Update on ultrasensitive technologies to facilitate research on blood biomarkers for central nervous system disorders. Alzheimer’s & Dementia: Diagnosis, Assessment & Disease Monitoring. 2016;3:98–102.
64.
Gendron TF, Chew J, Stankowski JN, Hayes LR, Zhang YJ, Prudencio M, Carlomagno Y, Daughrity LM, Jansen-West K, Perkerson EA, O’Raw A, Cook C, Pregent L, Belzil V, van Blitterswijk M, Tabassian LJ, Lee CW, Yue M, Tong J, Song Y, Castanedes-Casey M, Rousseau L, Phillips V, Dickson DW, Rademakers R, Fryer JD, Rush BK, Pedraza O, Caputo AM, Desaro P, Palmucci C, Robertson A, Heckman MG, Diehl NN, Wiggs E, Tierney M, Braun L, Farren J, Lacomis D, Ladha S, Fournier CN, McCluskey LF, Elman LB, Toledo JB, McBride JD, Tiloca C, Morelli C, Poletti B, Solca F, Prelle A. Poly(GP) proteins are a useful pharmacodynamic marker for -associated amyotrophic lateral sclerosis. Science Translational Medicine. 2017 Mar 29;9(383).
65.
Vincent A. Unravelling the pathogenesis of myasthenia gravis. Nature Reviews Immunology. 2002 Oct;2(10):797–804.
66.
Jacobson L, Polizzi A, Morriss-Kay G, Vincent A. Plasma from human mothers of fetuses with severe arthrogryposis multiplex congenita causes deformities in mice. Journal of Clinical Investigation. 1999 Apr 1;103(7):1031–1038.
67.
Viegas S, Jacobson L, Waters P, Cossins J, Jacob S, Leite MI, Webster R, Vincent A. Passive and active immunization models of MuSK-Ab positive myasthenia: Electrophysiological evidence for pre and postsynaptic defects. Experimental Neurology. 2012 Apr;234(2):506–512.
68.
Koneczny I, Cossins J, Vincent A. The role of muscle-specific tyrosine kinase (MuSK) and mystery of MuSK myasthenia gravis. Journal of Anatomy. 2014 Jan;224(1):29–35.
69.
Koneczny I, Cossins J, Waters P, Beeson D, Vincent A. MuSK Myasthenia Gravis IgG4 Disrupts the Interaction of LRP4 with MuSK but Both IgG4 and IgG1-3 Can Disperse Preformed Agrin-Independent AChR Clusters. PLoS ONE. 2013 Nov 7;8(11).
70.
Crisp SJ, Kullmann DM, Vincent A. Autoimmune synaptopathies. Nature Reviews Neuroscience. 2016 Feb;17(2):103–117.
71.
Woollacott IOC, Rohrer JD. The clinical spectrum of sporadic and familial forms of frontotemporal dementia. Journal of Neurochemistry. 2016 Aug;138:6–31.
72.
Gordon E, Rohrer JD, Fox NC. Advances in neuroimaging in frontotemporal dementia. Journal of Neurochemistry. 2016 Aug;138:193–210.
73.
Volume 58, Issue 3, March 2016. Volume 58, Issue 3, March 2016 [Internet]. Available from: https://link.springer.com/journal/12031/58/3
74.
Badders NM, Korff A, Miranda HC, Vuppala PK, Smith RB, Winborn BJ, Quemin ER, Sopher BL, Dearman J, Messing J, Kim NC, Moore J, Freibaum BD, Kanagaraj AP, Fan B, Tillman H, Chen PC, Wang Y, III BBF, Li Y, Kim HJ, La Spada AR, Taylor JP. Selective modulation of the androgen receptor AF2 domain rescues degeneration in spinal bulbar muscular atrophy. Nature Medicine. 2018 Mar 5;24(4):427–437.
75.
Beitel LK, Alvarado C, Mokhtar S, Paliouras M, Trifiro M. Mechanisms Mediating Spinal and Bulbar Muscular Atrophy: Investigations into Polyglutamine-Expanded Androgen Receptor Function and Dysfunction. Frontiers in Neurology. 2013;4.
76.
Cortes CJ, Ling SC, Guo LT, Hung G, Tsunemi T, Ly L, Tokunaga S, Lopez E, Sopher BL, Bennett CF, Shelton GD, Cleveland DW, La Spada AR. Muscle Expression of Mutant Androgen Receptor Accounts for Systemic and Motor Neuron Disease Phenotypes in Spinal and Bulbar Muscular Atrophy. Neuron. 2014 Apr;82(2):295–307.
77.
Fratta P, Nirmalananthan N, Masset L, Skorupinska I, Collins T, Cortese A, Pemble S, Malaspina A, Fisher EMC, Greensmith L, Hanna MG. Correlation of clinical and molecular features in spinal bulbar muscular atrophy. Neurology. 2014 Jun 10;82(23):2077–2084.
78.
Lieberman AP, Yu Z, Murray S, Peralta R, Low A, Guo S, Yu XX, Cortes CJ, Bennett CF, Monia BP, La Spada AR, Hung G. Peripheral Androgen Receptor Gene Suppression Rescues Disease in Mouse Models of Spinal and Bulbar Muscular Atrophy. Cell Reports. 2014 May;7(3):774–784.
79.
Malik B, Nirmalananthan N, Bilsland LG, La Spada AR, Hanna MG, Schiavo G, Gallo JM, Greensmith L. Absence of disturbed axonal transport in spinal and bulbar muscular atrophy. Human Molecular Genetics. 2011 May 1;20(9):1776–1786.
80.
Malik B, Nirmalananthan N, Gray AL, La Spada AR, Hanna MG, Greensmith L. Co-induction of the heat shock response ameliorates disease progression in a mouse model of human spinal and bulbar muscular atrophy: implications for therapy. Brain. 2013 Mar;136(3):926–943.
81.
Manzano R, Sorarú G, Grunseich C, Fratta P, Zuccaro E, Pennuto M, Rinaldi C. Beyond motor neurons: expanding the clinical spectrum in Kennedy’s disease. Journal of Neurology, Neurosurgery & Psychiatry. 2018 Aug;89(8):808–812.
82.
Milioto C, Malena A, Maino E, Polanco MJ, Marchioretti C, Borgia D, Pereira MG, Blaauw B, Lieberman AP, Venturini R, Plebani M, Sambataro F, Vergani L, Pegoraro E, Sorarù G, Pennuto M. Beta-agonist stimulation ameliorates the phenotype of spinal and bulbar muscular atrophy mice and patient-derived myotubes. Scientific Reports. 2017 Dec;7(1).
