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03/15/19 CLNE0020: Motoneurons, Neuromuscular Junctions and Associated Disease

| University College London

CLNE0020: Motoneurons, Neuromuscular View Online

Junctions and Associated Disease
(Academic Year 2018/19)

91 items

Drug development for ALS and other neurodegenerative diseases:

pharmacology perspective - Whiting (4 items)
Current and prospective disease-modifying therapies for amyotrophic lateral sclerosis -
John A Morren, Nestor Galvez-Jimenez, 2012-03

Clinical trials in amyotrophic lateral sclerosis: why so many negative trials and how can
trials be improved? - Hiroshi Mitsumoto, Benjamin R Brooks, Vincenzo Silani, 2014-11

What can we learn from the edaravone development program for ALS? - Nicholas J.
Maragakis, 2017-10-31

Principles of early drug discovery - JP Hughes, S Rees, SB Kalindjian, KL Philpott, 2011-03


Physiotherapy in paediatric diseases of motor neurons and

neuromuscular junctions: SMA and juvenile Myasthenia Gravis +
Physiotherapy in adult motor neuron diseases and Myasthenias -
Reznik (22 items)


Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and

neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management
- David J Birnkrant, Katharine Bushby, Carla M Bann, Susan D Apkon, Angela Blackwell,
David Brumbaugh, Laura E Case, Paula R Clemens, Stasia Hadjiyannakis, Shree Pandya,
Natalie Street, Jean Tomezsko, Kathryn R Wagner, Leanne M Ward, David R Weber,

Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac,

bone health, and orthopaedic management - David J Birnkrant, Katharine Bushby, Carla M
Bann, Benjamin A Alman, Susan D Apkon, Angela Blackwell, Laura E Case, Linda Cripe,
Stasia Hadjiyannakis, Aaron K Olson, Daniel W Sheehan, Julie Bolen, David R Weber,
Leanne M Ward, 2018-04

03/15/19 CLNE0020: Motoneurons, Neuromuscular Junctions and Associated Disease
| University College London


Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care,

emergency management, psychosocial care, and transitions of care across the lifespan -
David J Birnkrant, Katharine Bushby, Carla M Bann, Susan D Apkon, Angela Blackwell, Mary
K Colvin, Linda Cripe, Adrienne R Herron, Annie Kennedy, Kathi Kinnett, James Naprawa,
Garey Noritz, James Poysky, Natalie Street, Christina J Trout, David R Weber, Leanne M
Ward, 2018-05


Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for

diagnosis, rehabilitation, orthopedic and nutritional care - Eugenio Mercuri, Richard S.
Finkel, Francesco Muntoni, Brunhilde Wirth, Jacqueline Montes, Marion Main, Elena S.
Mazzone, Michael Vitale, Brian Snyder, Susana Quijano-Roy, Enrico Bertini, Rebecca Hurst
Davis, Oscar H. Meyer, Anita K. Simonds, Mary K. Schroth, Robert J. Graham, Janbernd
Kirschner, Susan T. Iannaccone, Thomas O. Crawford, Simon Woods, Ying Qian, Thomas
Sejersen, Francesco Muntoni, Brunhilde Wirth, Francesco Danilo Tiziano, Janbernd
Kirschner, Eduardo Tizzano, Haluk Topaloglu, Kathy Swoboda, Nigel Laing, Saito Kayoko,
Thomas Prior, Wendy K. Chung, Shou-Mei Wu, Jacqueline Montes, Elena Mazzone, Marion
Main, Caron Coleman, Richard Gee, Allan Glanzman, Anna-Karin Kroksmark, Kristin
Krosschell, Leslie Nelson, Kristy Rose, Agnieszka Stępień, Carole Vuillerot, Michael Vitale,
Brian Snyder, Susana Quijano-Roy, Jean Dubousset, , , , , , , , , , , , , , , , , , , , , , , 2018-02

Diagnosis and management of spinal muscular atrophy: Part 2: Pulmonary and acute care;
medications, supplements and immunizations; other organ systems; and ethics - Richard
S. Finkel, Eugenio Mercuri, Oscar H. Meyer, Anita K. Simonds, Mary K. Schroth, Robert J.
Graham, Janbernd Kirschner, Susan T. Iannaccone, Thomas O. Crawford, Simon Woods,
Francesco Muntoni, Brunhilde Wirth, Jacqueline Montes, Marion Main, Elena S. Mazzone,
Michael Vitale, Brian Snyder, Susana Quijano-Roy, Enrico Bertini, Rebecca Hurst Davis,
Ying Qian, Thomas Sejersen, 2018-03

Therapeutic approaches for spinal muscular atrophy (SMA) - M Scoto, R S Finkel, E Mercuri,
F Muntoni, 2017-9

Revised Hammersmith Scale for spinal muscular atrophy: A SMA specific clinical outcome
assessment tool - Danielle Ramsey, Mariacristina Scoto, Anna Mayhew, Marion Main, Elena
S. Mazzone, Jacqueline Montes, Roberto de Sanctis, Sally Dunaway Young, Rachel Salazar,
Allan M. Glanzman, Amy Pasternak, Janet Quigley, Elizabeth Mirek, Tina Duong, Richard
Gee, Matthew Civitello, Gihan Tennekoon, Marika Pane, Maria Carmela Pera, Kate Bushby,
John Day, Basil T. Darras, Darryl De Vivo, Richard Finkel, Eugenio Mercuri, Francesco
Muntoni, 2017-2-21

Revised upper limb module for spinal muscular atrophy: Development of a new module -
Elena S. Mazzone, Anna Mayhew, Jacqueline Montes, Danielle Ramsey, Lavinia Fanelli,
Sally Dunaway Young, Rachel Salazar, Roberto De Sanctis, Amy Pasternak, Allan

03/15/19 CLNE0020: Motoneurons, Neuromuscular Junctions and Associated Disease
| University College London

Glanzman, Giorgia Coratti, Matthew Civitello, Nicola Forcina, Richard Gee, Tina Duong,
Marika Pane, Mariacristina Scoto, Maria Carmela Pera, Sonia Messina, Gihan Tennekoon,
John W. Day, Basil T. Darras, Darryl C. De Vivo, Richard Finkel, Francesco Muntoni, Eugenio
Mercuri, 2017-06

MYESTHENIA GRAVIS (Juvenile and Adult)

The impact of physical exercise on neuromuscular function in Myasthenia gravis patients -

Elisabet Westerberg, Carl Johan Molin, Sören Spörndly Nees, Johan Widenfalk, Anna
Rostedt Punga, 2018-08

Pediatric Myasthenia Gravis - Jason H. Peragallo, 2017-05



Prevalence and orthopedic management of foot and ankle deformities in

Charcot-Marie-Tooth disease - Matilde Laurá, Dishan Singh, Gita Ramdharry, Jasper
Morrow, Mariola Skorupinska, Davide Pareyson, Joshua Burns, Richard A. Lewis, Steven S.
Scherer, David N. Herrmann, Nicholas Cullen, Christopher Bradish, Luca Gaiani, Nicolò
Martinelli, Paul Gibbons, Glenn Pfeffer, Phinit Phisitkul, Keith Wapner, James Sanders, Sam
Flemister, Michael E. Shy, Mary M. Reilly, 2018-02

221st ENMC International Workshop: - Mary M. Reilly, Davide Pareyson, Joshua Burns,
Matilde Laurá, Michael E. Shy, Dishan Singh, Per Henrik Agren, Viola Altmann, Jonathan
Baets, Peter Briggs, Joshua Burns, Karen Butcher, Luca Gaiani, Filippo Genovese, Paul
Gibbons, Matilde Laurá, Jan Willem Louwerens, Adnan Manzur, Isabella Moroni, Nicolò
Martinelli, Davide Pareyson, Glenn Pfeffer, Gita Ramdharry, Mary M. Reilly, Michael Shy,
Dishan Singh, Marco van der Linden, Wolfram Wenz, 2017-12

A pilot study of proximal strength training in Charcot-Marie-Tooth disease - Gita M.

Ramdharry, Alexander Pollard, Cheryl Anderson, Matilde Laurá, Sinead M. Murphy,
Magdalena Dudziec, Elizabeth L. Dewar, Elspeth Hutton, Robert Grant, Mary M. Reilly,


Amyotrophic lateral sclerosis: clinical perspectives - Summer Gibson, Verena Haringer,


Identifying who will benefit from non-invasive ventilation in amyotrophic lateral

sclerosis/motor neurone disease in a clinical cohort - David J Berlowitz, Mark E Howard,
Julio F Fiore, Stephen Vander Hoorn, Fergal J O'Donoghue, Justine Westlake, Anna Smith,
Fiona Beer, Susan Mathers, Paul Talman, 2016-03

03/15/19 CLNE0020: Motoneurons, Neuromuscular Junctions and Associated Disease
| University College London

Clinical aspects of motor neurone disease - Ceryl A. Harwood, Christopher J. McDermott,

Pamela J. Shaw, 2012-10

The value of muscle exercise in patients with amyotrophic lateral sclerosis - Vivian E.
Drory, Evgeny Goltsman, Jacqueline Goldman Reznik, Amnon Mosek, Amos D. Korczyn,

Pathomechanisms of Motoneuron disease - Kalmar (6 items)

Gene discovery in amyotrophic lateral sclerosis: implications for clinical management -
Ammar Al-Chalabi, Leonard H. van den Berg, Jan Veldink, 2017-2

Pathways to mitochondrial dysfunction in ALS pathogenesis - Maria Teresa Carrì, Nadia

D’Ambrosi, Mauro Cozzolino, 2017-02

Amyotrophic Lateral Sclerosis Pathogenesis Converges on Defects in Protein Homeostasis

Associated with TDP-43 Mislocalization and Proteasome-Mediated Degradation Overload -
G. Lin, D. Mao, H.J. Bellen

Stress granules at the intersection of autophagy and ALS - Zachary Monahan, Frank
Shewmaker, Udai Bhan Pandey, 2016-10

Proteostasis impairment in ALS - Céline Ruegsegger, Smita Saxena, 2016-10


State of play in amyotrophic lateral sclerosis genetics - Alan E Renton, Adriano Chiò, Bryan
J Traynor, 2014-1

Development and Maturation of Motor Neurons and Motor Units -

Bryson (8 items)
Neuronal specification in the spinal cord: inductive signals and transcriptional codes -
Thomas M. Jessell, 2000-10
Article | Recommended

Neural induction - Richard Harland, 2000-8


Chapter Six Hox Networks and the Origins of Motor Neuron Diversity - Jeremy S. Dasen,
Thomas M. Jessell
Chapter | Recommended

03/15/19 CLNE0020: Motoneurons, Neuromuscular Junctions and Associated Disease
| University College London

Motor Axon Pathfinding - D. Bonanomi, S. L. Pfaff, 2010-03-01

Article | Recommended

Neuromuscular synaptogenesis: coordinating partners with multiple functions - Houssam

Darabid, Anna P. Perez-Gonzalez, Richard Robitaille, 2014-11

Motor Neuron Diversity in Development and Disease - Kevin C. Kanning, Artem Kaplan,
Christopher E. Henderson, 2010-06
Article | Recommended

Assembly of Motor Circuits in the Spinal Cord: Driven to Function by Genetic and
Experience-Dependent Mechanisms - David R. Ladle, Eline Pecho-Vrieseling, Silvia Arber,
Article | Recommended

Spinal interneurons providing input to the final common path during locomotion - Robert
M. Brownstone, Tuan V. Bui

Embryonic and Postnatal Development of Neuromuscular Junctions -

Ross (9 items)

General but detailed review, covering most topics:

Neuromuscular Junction Formation, Aging, and Disorders - Lei Li, Wen-Cheng Xiong, Lin Mei
, 2018-02-10


Role of extracellular matrix proteins and their receptors in the development of the
vertebrate neuromuscular junction - Neha Singhal, Paul T. Martin, 2011-11

Laminins promote postsynaptic maturation by an autocrine mechanism at the

neuromuscular junction - Hiroshi Nishimune, Gregorio Valdez, George Jarad, Casey L.
Moulson, Ulrich Müller, Jeffrey H. Miner, Joshua R. Sanes, 2008-09-22


Degeneration of Neuromuscular Junction in Age and Dystrophy - Rüdiger Rudolf, Muzamil

Majid Khan, Siegfried Labeit, Michael R. Deschenes, 2014-05-22

03/15/19 CLNE0020: Motoneurons, Neuromuscular Junctions and Associated Disease
| University College London


The human NMJ

Cellular and Molecular Anatomy of the Human Neuromuscular Junction - Ross A. Jones, Carl
Harrison, Samantha L. Eaton, Maica Llavero Hurtado, Laura C. Graham, Leena
Alkhammash, Oladayo A. Oladiran, Andy Gale, Douglas J. Lamont, Hamish Simpson, Martin
W. Simmen, Christian Soeller, Thomas M. Wishart, Thomas H. Gillingwater, 2017-11

Congenital myasthenic syndromes - Munot (6 items)

Clinical and research strategies for limb-girdle congenital myasthenic syndromes - Emily
O'Connor, Ana Töpf, René P. Zahedi, Sally Spendiff, Daniel Cox, Andreas Roos, Hanns
Lochmüller, 2018-01

Congenital myasthenic syndromes: pathogenesis, diagnosis, and treatment - Andrew G

Engel, Xin-Ming Shen, Duygu Selcen, Steven M Sine, 2015-04

Congenital myasthenic syndromes and the neuromuscular junction - Pedro M. Rodríguez

Cruz, Jacqueline Palace, David Beeson, 2014-10

Inherited disorders of the neuromuscular junction: an update - Pedro M. Rodríguez Cruz,

Jacqueline Palace, David Beeson, 2014-11

Mutations in

cause congenital myasthenic syndrome and bridge myasthenic disorders with

dystroglycanopathies - Katsiaryna Belaya, Pedro M. Rodríguez Cruz, Wei Wei Liu, Susan
Maxwell, Simon McGowan, Maria E. Farrugia, Richard Petty, Timothy J. Walls, Maryam
Sedghi, Keivan Basiri, Wyatt W. Yue, Anna Sarkozy, Marta Bertoli, Matthew Pitt, Robin
Kennett, Andrew Schaefer, Kate Bushby, Matt Parton, Hanns Lochmüller, Jacqueline Palace,
Francesco Muntoni, David Beeson, 2015-09

Congenital myopathies with secondary neuromuscular transmission defects; A case report

and review of the literature - Pedro M. Rodríguez Cruz, Caroline Sewry, David Beeson,
Sandeep Jayawant, Waney Squier, Robert McWilliam, Jacqueline Palace, 2014-12

Myasthenia gravis: Lambert Eaton Myasthenic syndromes and

botulinism - Kullman (8 items)

03/15/19 CLNE0020: Motoneurons, Neuromuscular Junctions and Associated Disease
| University College London

Autoimmune synaptopathies - Sarah J. Crisp, Dimitri M. Kullmann, Angela Vincent, 2016-2


Myasthenia Gravis - Nils E. Gilhus, 2016-12-29


Myasthenia Gravis - Linda L. Kusner, Henry J. Kaminski


Other Proven and Putative Autoimmune Disorders of the Peripheral Nervous System - Doris
G. Leung, 2017-03

Myasthenia and related disorders of the neuromuscular junction - J. Spillane, D. J. Beeson,

D. M. Kullmann, 2010-08-01

Autoimmune myasthenia gravis: emerging clinical and biological heterogeneity - Matthew

N Meriggioli, Donald B Sanders, 2009-05

Lambert-Eaton syndrome IgG inhibits transmitter release via P/Q Ca2+ channels - J.
Spillane, Y. Ermolyuk, M. Cano-Jaimez, B. Lang, A. Vincent, K. E. Volynski, D. M. Kullmann,

Randomized Trial of Thymectomy in Myasthenia Gravis - Gil I. Wolfe, Henry J. Kaminski,

Inmaculada B. Aban, Greg Minisman, Hui-Chien Kuo, Alexander Marx, Philipp Ströbel,
Claudio Mazia, Joel Oger, J. Gabriel Cea, Jeannine M. Heckmann, Amelia Evoli, Wilfred Nix,
Emma Ciafaloni, Giovanni Antonini, Rawiphan Witoonpanich, John O. King, Said R.
Beydoun, Colin H. Chalk, Alexandru C. Barboi, Anthony A. Amato, Aziz I. Shaibani, Bashar
Katirji, Bryan R.F. Lecky, Camilla Buckley, Angela Vincent, Elza Dias-Tosta, Hiroaki
Yoshikawa, Márcia Waddington-Cruz, Michael T. Pulley, Michael H. Rivner, Anna
Kostera-Pruszczyk, Robert M. Pascuzzi, Carlayne E. Jackson, Guillermo S. Garcia Ramos,
Jan J.G.M. Verschuuren, Janice M. Massey, John T. Kissel, Lineu C. Werneck, Michael
Benatar, Richard J. Barohn, Rup Tandan, Tahseen Mozaffar, Robin Conwit, Joanne
Odenkirchen, Joshua R. Sonett, Alfred Jaretzki, John Newsom-Davis, Gary R. Cutter,

Clinical features of Motor neuron disease - Orrell (5 items)

Diagnosis and management of motor neurone disease - Orrell, Richard WBarclay, Chris,
Sep 2016

Pathogenesis of amyotrophic lateral sclerosis - Sarah Morgan, Richard W. Orrell, 2016-09


Neurology: an illustrated colour text - Geraint Fuller, Mark Manford, 2010

03/15/19 CLNE0020: Motoneurons, Neuromuscular Junctions and Associated Disease
| University College London

Book | Disorders of the Motor Neurone (pag. 108)

Motor neurone disease: assessment and management | Guidance and guidelines | NICE

Epidemiology of amyotrophic lateral sclerosis: A review of literature - P. Couratier, P.

Corcia, G. Lautrette, M. Nicol, P.-M. Preux, B. Marin, 2016-01

Biomarkers in MND - Lu (5 items)

Roadmap and standard operating procedures for biobanking and discovery of
neurochemical markers in ALS - Markus Otto, Robert Bowser, Martin Turner, James Berry,
Johannes Brettschneider, James Connor, Júlia Costa, Merit Cudkowicz, Jonathan Glass, Olaf
Jahn, Stefan Lehnert, Andrea Malaspina, Lucilla Parnetti, Axel Petzold, Pamela Shaw,
Alexander Sherman, Petra Steinacker, Sigurd Süßmuth, Charlotte Teunissen, Hayrettin
Tumani, Anna Wuolikainen, Albert Ludolph, 2012-01

Neurofilament light chain: A prognostic biomarker in amyotrophic lateral sclerosis - C.-H.

Lu, C. Macdonald-Wallis, E. Gray, N. Pearce, A. Petzold, N. Norgren, G. Giovannoni, P.
Fratta, K. Sidle, M. Fish, R. Orrell, R. Howard, K. Talbot, L. Greensmith, J. Kuhle, M. R.
Turner, A. Malaspina, 2015-06-02

ALS biomarkers for therapy development: State of the field and future directions - Michael
Benatar, Kevin Boylan, Andreas Jeromin, Seward B. Rutkove, James Berry, Nazem Atassi,
Lucie Bruijn, 2016-02

Update on ultrasensitive technologies to facilitate research on blood biomarkers for central

nervous system disorders - Ulf Andreasson, Kaj Blennow, Henrik Zetterberg, 2016

Poly(GP) proteins are a useful pharmacodynamic marker for

-associated amyotrophic lateral sclerosis - Tania F. Gendron, Jeannie Chew,

Jeannette N. Stankowski, Lindsey R. Hayes, Yong-Jie Zhang, Mercedes Prudencio, Yari
Carlomagno, Lillian M. Daughrity, Karen Jansen-West, Emilie A. Perkerson, Aliesha O’Raw,
Casey Cook, Luc Pregent, Veronique Belzil, Marka van Blitterswijk, Lilia J. Tabassian, Chris
W. Lee, Mei Yue, Jimei Tong, Yuping Song, Monica Castanedes-Casey, Linda Rousseau,
Virginia Phillips, Dennis W. Dickson, Rosa Rademakers, John D. Fryer, Beth K. Rush, Otto
Pedraza, Ana M. Caputo, Pamela Desaro, Carla Palmucci, Amelia Robertson, Michael G.
Heckman, Nancy N. Diehl, Edythe Wiggs, Michael Tierney, Laura Braun, Jennifer Farren,
David Lacomis, Shafeeq Ladha, Christina N. Fournier, Leo F. McCluskey, Lauren B. Elman,
Jon B. Toledo, Jennifer D. McBride, Cinzia Tiloca, Claudia Morelli, Barbara Poletti, Federica
Solca, Alessandro Prelle, , , , , , , , , , , , , , , , , , , , , , , , , , , , , , 2017-03-29

03/15/19 CLNE0020: Motoneurons, Neuromuscular Junctions and Associated Disease
| University College London

Immunology of the neuromuscular junction; Acquired diseases of

Neuromuscular Junctions - Vincent (6 items)
Unravelling the pathogenesis of myasthenia gravis - Angela Vincent, 2002-10

Plasma from human mothers of fetuses with severe arthrogryposis multiplex congenita
causes deformities in mice - Leslie Jacobson, Agata Polizzi, Gillian Morriss-Kay, Angela
Vincent, 1999-4-1

Passive and active immunization models of MuSK-Ab positive myasthenia:

Electrophysiological evidence for pre and postsynaptic defects - Stuart Viegas, Leslie
Jacobson, Patrick Waters, Judith Cossins, Saiju Jacob, M. Isabel Leite, Richard Webster,
Angela Vincent, 2012-04

The role of muscle-specific tyrosine kinase (MuSK) and mystery of MuSK myasthenia gravis
- Inga Koneczny, Judith Cossins, Angela Vincent, 2014-01

MuSK Myasthenia Gravis IgG4 Disrupts the Interaction of LRP4 with MuSK but Both IgG4
and IgG1-3 Can Disperse Preformed Agrin-Independent AChR Clusters - Inga Koneczny,
Judith Cossins, Patrick Waters, David Beeson, Angela Vincent, 2013-11-7

Autoimmune synaptopathies - Sarah J. Crisp, Dimitri M. Kullmann, Angela Vincent, 2016-2


ALS-FTD - Rohrer (2 items)

The clinical spectrum of sporadic and familial forms of frontotemporal dementia - Ione O.
C. Woollacott, Jonathan D. Rohrer, 2016-08

Advances in neuroimaging in frontotemporal dementia - Elizabeth Gordon, Jonathan D.

Rohrer, Nick C. Fox, 2016-08

Spinobulbar Muscular Atrophy (SBMA) - Malik (10 items)

Volume 58, Issue 3, March 2016

Selective modulation of the androgen receptor AF2 domain rescues degeneration in spinal
bulbar muscular atrophy - Nisha M Badders, Ane Korff, Helen C Miranda, Pradeep K
Vuppala, Rebecca B Smith, Brett J Winborn, Emmanuelle R Quemin, Bryce L Sopher,
Jennifer Dearman, James Messing, Nam Chul Kim, Jennifer Moore, Brian D Freibaum,
Anderson P Kanagaraj, Baochang Fan, Heather Tillman, Ping-Chung Chen, Yingzhe Wang,

03/15/19 CLNE0020: Motoneurons, Neuromuscular Junctions and Associated Disease
| University College London

Burgess B Freeman III, Yimei Li, Hong Joo Kim, Albert R La Spada, J Paul Taylor, 2018-3-5

Mechanisms Mediating Spinal and Bulbar Muscular Atrophy: Investigations into

Polyglutamine-Expanded Androgen Receptor Function and Dysfunction - Lenore K. Beitel,
Carlos Alvarado, Shaza Mokhtar, Miltiadis Paliouras, Mark Trifiro, 2013

Muscle Expression of Mutant Androgen Receptor Accounts for Systemic and Motor Neuron
Disease Phenotypes in Spinal and Bulbar Muscular Atrophy - Constanza J. Cortes,
Shuo-Chien Ling, Ling T. Guo, Gene Hung, Taiji Tsunemi, Linda Ly, Seiya Tokunaga, Edith
Lopez, Bryce L. Sopher, C. Frank Bennett, G. Diane Shelton, Don W. Cleveland, Albert R.
La Spada, 2014-04

Correlation of clinical and molecular features in spinal bulbar muscular atrophy - P. Fratta,
N. Nirmalananthan, L. Masset, I. Skorupinska, T. Collins, A. Cortese, S. Pemble, A.
Malaspina, E. M. C. Fisher, L. Greensmith, M. G. Hanna, 2014-06-10

Peripheral Androgen Receptor Gene Suppression Rescues Disease in Mouse Models of

Spinal and Bulbar Muscular Atrophy - Andrew P. Lieberman, Zhigang Yu, Sue Murray,
Raechel Peralta, Audrey Low, Shuling Guo, Xing Xian Yu, Constanza J. Cortes, C. Frank
Bennett, Brett P. Monia, Albert R. La Spada, Gene Hung, 2014-05

Absence of disturbed axonal transport in spinal and bulbar muscular atrophy - B. Malik, N.
Nirmalananthan, L. G. Bilsland, A. R. La Spada, M. G. Hanna, G. Schiavo, J.-M. Gallo, L.
Greensmith, 2011-05-01

Co-induction of the heat shock response ameliorates disease progression in a mouse

model of human spinal and bulbar muscular atrophy: implications for therapy - Bilal Malik,
Niranjanan Nirmalananthan, Anna L. Gray, Albert R. La Spada, Michael G. Hanna, Linda
Greensmith, 2013-03

Beyond motor neurons: expanding the clinical spectrum in Kennedy’s disease - Raquel
Manzano, Gianni Sorarú, Christopher Grunseich, Pietro Fratta, Emanuela Zuccaro, Maria
Pennuto, Carlo Rinaldi, 2018-08

Beta-agonist stimulation ameliorates the phenotype of spinal and bulbar muscular atrophy
mice and patient-derived myotubes - Carmelo Milioto, Adriana Malena, Eleonora Maino,
Maria J. Polanco, Caterina Marchioretti, Doriana Borgia, Marcelo Gomes Pereira, Bert
Blaauw, Andrew P. Lieberman, Roberta Venturini, Mario Plebani, Fabio Sambataro,
Lodovica Vergani, Elena Pegoraro, Gianni Sorarù, Maria Pennuto, 2017-12