Michella Guglieri

Affiliations
  • John Walton Muscular Dystrophy Research Centre Newcastle University Translational and Clinical Research Institute and Newcastle Hospitals NHS Foundation Trust Newcastle upon Tyne United Kingdom.

Publications (67)

Fractures and Linear Growth in a Nationwide Cohort of Boys With Duchenne Muscular Dystrophy With and Without Glucocorticoid Treatment: Results From the UK NorthStar Database.

JAMA neurology
Avec: Shuko Joseph , Cunyi Wang , Kate Bushby , Michaela Guglieri , Iain Horrocks , Volker Straub , S Faisal Ahmed , Sze Choong Wong , Auteur inconnu

Preventing Cardiomyopathy in DMD: A Randomized Placebo-Controlled Drug Trial.

Neurology. Clinical practice
Avec: John P Bourke , Gillian Watson , Stefan Spinty , Andrew Bryant , Helen Roper , Thomas Chadwick , Ruth Wood , Elaine McColl , Kate Bushby , Francesco Muntoni , Michela Guglieri , Auteur inconnu

Pubertal induction in adolescents with DMD is associated with high satisfaction, gonadotropin release and increased muscle contractile surface area.

European journal of endocrinology
Avec: Claire L Wood , Kieren G Hollingsworth , Eric Hughes , Sadhanandham Punniyakodi , Robert Muni-Lofra , Anna Mayhew , Rod T Mitchell , Michela Guglieri , Timothy D Cheetham , Volker Straub

Multi-Omics Identifies Circulating miRNA and Protein Biomarkers for Facioscapulohumeral Dystrophy.

Journal of personalized medicine
Avec: Christopher R Heier , Aiping Zhang , Nhu Y Nguyen , Christopher B Tully , Aswini Panigrahi , Heather Gordish-Dressman , Sachchida Nand Pandey , Michela Guglieri , Monique M Ryan , Paula R Clemens , Mathula Thangarajh , Richard Webster , Edward C Smith , Anne M Connolly , Craig M McDonald , Peter Karachunski , Mar Tulinius , Amy Harper , Jean K Mah , Alyson A Fiorillo , Yi-Wen Chen , Cooperative International Neuromuscular Research Group Cinrg Investigators

Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy.

Neuromuscular disorders : NMD
Avec: Craig Campbell , Elaine McColl , Michael P McDermott , William B Martens , Michela Guglieri , Robert C Griggs , Auteur inconnu

Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.

JAMA neurology
Avec: Michela Guglieri , Paula R Clemens , Seth J Perlman , Edward C Smith , Iain Horrocks , Richard S Finkel , Jean K Mah , Nicolas Deconinck , Nathalie Goemans , Jana Haberlova , Volker Straub , Laurel J Mengle-Gaw , Benjamin D Schwartz , Amy D Harper , Perry B Shieh , Liesbeth De Waele , Diana Castro , Michelle L Yang , Monique M Ryan , Craig M McDonald , Mar Tulinius , Richard Webster , Hugh J McMillan , Nancy L Kuntz , Vashmi K Rao , Giovanni Baranello , Stefan Spinty , Anne-Marie Childs , Annie M Sbrocchi , Kathryn A Selby , Migvis Monduy , Yoram Nevo , Juan J Vilchez-Padilla , Andres Nascimento-Osorio , Erik H Niks , Imelda J M de Groot , Marina Katsalouli , Meredith K James , Johannes van den Anker , Jesse M Damsker , Alexandra Ahmet , Leanne M Ward , Mark Jaros , Phil Shale , Utkarsh J Dang , Eric P Hoffman

Can symptomatic nmDuchenne carriers benefit from treatment with ataluren? Results of 193-month follow-up.

Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology
Avec: Amir Dori , Michela Guglieri , Marianna Scutifero , Luigia Passamano , Antonio Trabacca , Luisa Politano

Natural history of Duchenne muscular dystrophy in the United Kingdom: A descriptive study using the Clinical Practice Research Datalink.

Brain and behavior
Avec: Jonathan Broomfield , Keith Abrams , Nick Latimer , Michela Guglieri , Mark Rutherford , Michael Crowther

Neurology
Avec: Francesco Muntoni , James Signorovitch , Gautam Sajeev , Henry Lane , Madeline Jenkins , Ibrahima Dieye , Susan J Ward , Craig McDonald , Nathalie Goemans , Erik H Niks , Brenda Wong , Laurent Servais , Volker Straub , Michela Guglieri , Imelda J M de Groot , Mary Chesshyre , Cuixia Tian , Adnan Y Manzur , Eugenio Mercuri , Annemieke Aartsma-Rus , Auteur inconnu

Beyond pGALS: the need for a multifaceted musculoskeletal decision-making tool ('pGALSplus') in community-based clinical practice.

Rheumatology advances in practice
Avec: Vicky Mercer , Nicola Smith , Sharmila Jandial , Michela Guglieri , Simon A Jones , Helen E Foster

Assessing Pharmacogenomic loci Associated with the Pharmacokinetics of Vamorolone in Boys with Duchenne Muscular Dystrophy.

Journal of clinical pharmacology
Avec: Xiaonan Li , Daniele Sabbatini , Elena Pegoraro , Luca Bello , Paula Clemens , Michela Guglieri , John van den Anker , Jesse Damsker , John McCall , Utkarsh J Dang , Eric P Hoffman , William J Jusko

Respiratory and upper limb function as outcome measures in ambulant and non-ambulant subjects with Duchenne muscular dystrophy: A prospective multicentre study.

Neuromuscular disorders : NMD
Avec: V Ricotti , V Selby , D Ridout , J Domingos , V Decostre , A Mayhew , M Eagle , J Butler , M Guglieri , M Van der Holst , M Jansen , J J G M Verschuuren , I J M de Groot , E H Niks , L Servais , V Straub , T Voit , J Y Hogrel , F Muntoni

Development of a New Quality of Life Measure for Duchenne Muscular Dystrophy Using Mixed Methods: The DMD-QoL.

Neurology
Avec: Philip A Powell , Jill Carlton , Donna Rowen , Fleur Chandler , Michela Guglieri , John E Brazier

The impact of testosterone therapy on quality of life in adolescents with Duchenne muscular dystrophy.

Neuromuscular disorders : NMD
Avec: C L Wood , J Page , J Foggin , M Guglieri , V Straub , T D Cheetham

Clinical and genetic spectrum of a large cohort of patients with δ-sarcoglycan muscular dystrophy.

Brain : a journal of neurology
Avec: Jorge Alonso-Pérez , Lidia González-Quereda , Claudio Bruno , Chiara Panicucci , Afagh Alavi , Shahriar Nafissi , Yalda Nilipour , Edmar Zanoteli , Lucas Michielon de Augusto Isihi , Béla Melegh , Kinga Hadzsiev , Nuria Muelas , Juan J Vílchez , Mario Emilio Dourado , Naz Kadem , Gultekin Kutluk , Muhammad Umair , Muhammad Younus , Elena Pegorano , Luca Bello , Thomas O Crawford , Xavier Suárez-Calvet , Ana Töpf , Michela Guglieri , Chiara Marini-Bettolo , Pia Gallano , Volker Straub , Jordi Díaz-Manera

Clinical and Genetic Characteristics in Young, Glucocorticoid-Naive Boys With Duchenne Muscular Dystrophy.

Neurology
Avec: Marianela Schiava , Rachel Amos , Henriette VanRuiten , Michael P McDermott , Williams B Martens , Stephanie Gregory , Anna Mayhew , Elaine McColl , Rabi Tawil , Tracey Willis , Kate Bushby , Robert C Griggs , Michela Guglieri , Auteur inconnu

Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial.

JAMA network open
Avec: Jean K Mah , Paula R Clemens , Michela Guglieri , Edward C Smith , Richard S Finkel , Mar Tulinius , Yoram Nevo , Monique M Ryan , Richard Webster , Diana Castro , Nancy L Kuntz , Craig M McDonald , Jesse M Damsker , Benjamin D Schwartz , Laurel J Mengle-Gaw , Stefan Jackowski , Georgia Stimpson , Deborah A Ridout , Vandana Ayyar-Gupta , Giovanni Baranello , Adnan Y Manzur , Francesco Muntoni , Heather Gordish-Dressman , Mika Leinonen , Leanne M Ward , Eric P Hoffman , Utkarsh J Dang , Auteur inconnu

Functional outcome measures in young, steroid-naïve boys with Duchenne muscular dystrophy.

Neuromuscular disorders : NMD
Avec: Anna G Mayhew , Dionne Moat , Michael P McDermott , Michelle Eagle , Robert C Griggs , Wendy M King , Meredith K James , Robert Muni-Lofra , Alison Shillington , Sarah Gregson , Lindsey Pallant , Christy Skura , Loretta A Staudt , Katy Eichinger , Heather McMurchie , Rosanna Rabb , Marina Di Marco , Sarah Brown , Riccardo Zanin , Maria Teresa Arnoldi , Melissa McIntyre , Amelia Wilson , Lindsay N Alfano , Linda P Lowes , Colleen Blomgren , Evelin Milev , Mario Iodice , Amy Pasternak , Angela Chiu , Ilka Lehnert , Nicole Claus , Kathy A Dieruf , Enrica Rolle , Alina Nicorici , Barbara Andres , Elke Hobbiebrunken , Gerda Roetmann , Victoria Kern , Matthew Civitello , Sibylle Vogt , Melissa J Hayes , Cheryl Scholtes , Catherine Lacroix , Tara Gunn , Sinead Warner , Jennifer Newman , Andrea Barp , Katherine Kundrat , Staci Kovelman , Penny J Powers , Michela Guglieri , Auteur inconnu

Longitudinal Analysis of Respiratory Function of Different Types of Limb Girdle Muscular Dystrophies Reveals Independent Trajectories.

Neurology. Genetics
Avec: Robert Muni-Lofra , Eduard Juanola-Mayos , Marianela Schiava , Dionne Moat , Maha Elseed , Jassi Michel-Sodhi , Elizabeth Harris , Michelle McCallum , Ursula Moore , Mark Richardson , Christina Trainor , Karen Wong , Monika Malinova , Carla Bolano-Diaz , Michael John Keogh , Elisabetta Ghimenton , Jose Verdu-Diaz , Anna Mayhew , Michela Guglieri , Volker Straub , Meredith K James , Chiara Marini-Bettolo , Jordi Diaz-Manera

Association between neurodevelopmental impairments and motor function in Duchenne muscular dystrophy.

Annals of clinical and translational neurology
Avec: Mathula Thangarajh , Michael P McDermott , Michela Guglieri , Robert C Griggs

Is ongoing testosterone required after pubertal induction in Duchenne muscular dystrophy?

Endocrine connections
Avec: Claire L Wood , Kieren G Hollingsworth , Edrina Bokaie , Eric Hughes , Robert Muni-Lofra , Anna Mayhew , Rod T Mitchell , Michela Guglieri , Joseph McElvaney , Timothy D Cheetham , Volker Straub

Reference curves of motor function outcomes in young steroid-naïve males with Duchenne muscular dystrophy.

Developmental medicine and child neurology
Avec: Jasmine Hoskens , Marianela Schiava , Nathalie Goemans , Hilde Feys , Michael P McDermott , William B Martens , Anna Mayhew , Robert C Griggs , Katrijn Klingels , Michela Guglieri

Decoding the muscle transcriptome of patients with late onset Pompe disease reveals markers of disease progression.

Brain : a journal of neurology
Avec: Alexandra Monceau , Rasya Gokul Nath , Xavier Suárez-Calvet , Olimpia Musumeci , Antonio Toscano , Biruta Kierdaszuk , Anna Kostera-Pruszczyk , Cristina Domínguez-González , Aurelio Hernández-Lain , Carmen Paradas , Eloy Rivas , George Papadimas , Constantinos Papadopoulos , Margarita Chrysanthou-Piterou , Eduard Gallardo , Montse Olivé , James Lilleker , Mark E Roberts , Domenica Marchese , Giulia Lunazzi , Holger Heyn , Esther Fernández-Simón , Elisa Villalobos , James Clark , Panos Katsikis , Catherine Collins , Priyanka Mehra , Zoe Laidler , Amy Vincent , Giorgio Tasca , Chiara Marini-Bettolo , Michela Guglieri , Volker Straub , Nina Raben , Jordi Díaz-Manera

Adrenal suppression from vamorolone and prednisone in Duchenne muscular dystrophy: results from the phase 2b clinical trial.

The Journal of clinical endocrinology and metabolism
Avec: Alexandra Ahmet , Rebecca Tobin , Utkarsh J Dang , Raoul Rooman , Michela Guglieri , Paula R Clemens , Eric P Hoffman , Leanne M Ward

New genotype-phenotype correlations in a large European cohort of patients with sarcoglycanopathy.

Brain : a journal of neurology
Avec: Jorge Alonso-Pérez , Lidia González-Quereda , Luca Bello , Michela Guglieri , Volker Straub , Pia Gallano , Claudio Semplicini , Elena Pegoraro , Vittoria Zangaro , Andrés Nascimento , Carlos Ortez , Giacomo Pietro Comi , Leroy Ten Dam , Marianne De Visser , A J van der Kooi , Cristina Garrido , Manuela Santos , Ulrike Schara , Andrea Gangfuß , Nicoline Løkken , Jesper Helbo Storgaard , John Vissing , Benedikt Schoser , Gabriele Dekomien , Bjarne Udd , Johanna Palmio , Adele D'Amico , Luisa Politano , Vincenzo Nigro , Claudio Bruno , Chiara Panicucci , Anna Sarkozy , Omar Abdel-Mannan , Alicia Alonso-Jimenez , Kristl G Claeys , David Gomez-Andrés , Francina Munell , Laura Costa-Comellas , Jana Haberlová , Marie Rohlenová , De Vos Elke , Jan L De Bleecker , Cristina Dominguez-González , Giorgio Tasca , Claudia Weiss , Nicolas Deconinck , Roberto Fernández-Torrón , Adolfo López de Munain , Ana Camacho-Salas , Béla Melegh , Kinga Hadzsiev , Lea Leonardis , Blaz Koritnik , Matteo Garibaldi , Juan Carlos de Leon-Hernández , Edoardo Malfatti , Arturo Fraga-Bau , Isabelle Richard , Isabel Illa , Jordi Díaz-Manera

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.

Nucleic acid therapeutics
Avec: Laurent Servais , Eugenio Mercuri , Volker Straub , Michela Guglieri , Andreea M Seferian , Mariacristina Scoto , Daniela Leone , Erica Koenig , Navid Khan , Ashish Dugar , Xiaodong Wang , Baoguang Han , Dan Wang , Francesco Muntoni , Auteur inconnu

The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy.

Acta neuropathologica communications
Avec: Dominic Scaglioni , Francesco Catapano , Matthew Ellis , Silvia Torelli , Darren Chambers , Lucy Feng , Matthew Beck , Caroline Sewry , Mauro Monforte , Shawn Harriman , Erica Koenig , Jyoti Malhotra , Linda Popplewell , Michela Guglieri , Volker Straub , Eugenio Mercuri , Laurent Servais , Rahul Phadke , Jennifer Morgan , Francesco Muntoni

Miyoshi myopathy and limb girdle muscular dystrophy R2 are the same disease.

Neuromuscular disorders : NMD
Avec: Ursula Moore , Heather Gordish , Jordi Diaz-Manera , Meredith K James , Anna G Mayhew , Michela Guglieri , Roberto Fernandez-Torron , Laura E Rufibach , Jia Feng , Andrew M Blamire , Pierre G Carlier , Simone Spuler , John W Day , Kristi J Jones , Diana X Bharucha-Goebel , Emmanuelle Salort-Campana , Alan Pestronk , Maggie C Walter , Carmen Paradas , Tanya Stojkovic , Madoka Mori-Yoshimura , Elena Bravver , Elena Pegoraro , Linda Pax Lowes , Jerry R Mendell , Kate Bushby , Volker Straub , Auteur inconnu

A survey of the feasibility of developing osteoporosis clinical trials in Duchenne muscular dystrophy: Survey of the opinion of young people with Duchenne muscular dystrophy, families and clinicians.

Clinical trials (London, England)
Avec: Sze Choong Wong , Shuko Joseph , Nadia Capaldi , Marina Di Marco , Jennifer Dunne , Michela Guglieri , Iain Horrocks , Volker Straub , S Faisal Ahmed , Auteur inconnu

Can symptomatic nmDuchenne carriers benefit from treatment with ataluren? Results of 193-month follow-up.

Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology
Avec: Amir Dori , Michela Guglieri , Marianna Scutifero , Luigia Passamano , Antonio Trabacca , Luisa Politano

Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach.

Frontiers in neurology
Avec: Anna G Mayhew , Meredith K James , Ursula Moore , Helen Sutherland , Marni Jacobs , Jia Feng , Linda Pax Lowes , Lindsay N Alfano , Robert Muni Lofra , Laura E Rufibach , Kristy Rose , Tina Duong , Luca Bello , Irene Pedrosa-Hernández , Scott Holsten , Chikako Sakamoto , Aurélie Canal , Nieves Sánchez-Aguilera Práxedes , Simone Thiele , Catherine Siener , Bruno Vandevelde , Brittney DeWolf , Elke Maron , Heather Gordish-Dressman , Heather Hilsden , Michela Guglieri , Jean-Yves Hogrel , Andrew M Blamire , Pierre G Carlier , Simone Spuler , John W Day , Kristi J Jones , Diana X Bharucha-Goebel , Emmanuelle Salort-Campana , Alan Pestronk , Maggie C Walter , Carmen Paradas , Tanya Stojkovic , Madoka Mori-Yoshimura , Elena Bravver , Jordi Díaz-Manera , Elena Pegoraro , Jerry R Mendell , Volker Straub

Development of respiratory care guidelines for Duchenne muscular dystrophy in the UK: key recommendations for clinical practice.

Thorax
Avec: Anne-Marie Childs , Catherine Turner , Ronan Astin , Stephen Bianchi , John Bourke , Vicki Cunningham , Lisa Edel , Christopher Edwards , Phillippa Farrant , Jane Heraghty , Meredith James , Charlotte Massey , Ben Messer , Jassi Michel Sodhi , Patrick Brian Murphy , Marianela Schiava , Ajit Thomas , Federica Trucco , Michela Guglieri

Factors Associated With Early Motor Function Trajectories in DMD After Glucocorticoid Initiation: Post Hoc Analysis of the FOR-DMD Trial.

Neurology
Avec: Marianela Schiava , Michael P McDermott , Jonathan Broomfield , Keith R Abrams , Anna G Mayhew , Craig M McDonald , William B Martens , Stephanie J Gregory , Robert C Griggs , Michela Guglieri , Auteur inconnu

T Cell Responses to Dystrophin in a Natural History Study of Duchenne Muscular Dystrophy.

Human gene therapy
Avec: Karen Anthony , Pierpaolo Ala , Francesco Catapano , Jinhong Meng , Joana Domingos , Mark Perry , Valeria Ricotti , Kate Maresh , Lauren C Phillips , Laurent Servais , Andreea M Seferian , Silvana De Lucia , Imelda de Groot , Yvonne D Krom , J G M Verschuuren , Erik H Niks , Volker Straub , Michela Guglieri , Thomas Voit , Jennifer Morgan , Francesco Muntoni

Patient reported pregnancy and birth outcomes in genetic neuromuscular diseases.

Neuromuscular disorders : NMD
Avec: Ursula Moore , Sarah Shira Emmons , Laura Rufibach , V Straub , Jordi Diaz-Manera , Michela Guglieri

The development of pGALSplus: evaluating feasibility and acceptability of an assessment to facilitate the identification and triage of children with musculoskeletal presentations.

Rheumatology advances in practice
Avec: Vicky Mercer , Nicola Smith , Michela Guglieri , Simon A Jones , Jeremy R Parr , Helen E Foster , Sharmila Jandial

Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale.

Annals of neurology
Avec: Marni B Jacobs , Meredoith K James , Linda P Lowes , Lindsay N Alfano , Michelle Eagle , Robert Muni Lofra , Ursula Moore , Jia Feng , Laura E Rufibach , Kristy Rose , Tina Duong , Luca Bello , Irene Pedrosa-Hernández , Scott Holsten , Chikako Sakamoto , Aurélie Canal , Nieves Sanchez-Aguilera Práxedes , Simone Thiele , Catherine Siener , Bruno Vandevelde , Brittney DeWolf , Elke Maron , Michela Guglieri , Jean-Yves Hogrel , Andrew M Blamire , Pierre G Carlier , Simone Spuler , John W Day , Kristi J Jones , Diana X Bharucha-Goebel , Emmanuelle Salort-Campana , Alan Pestronk , Maggie C Walter , Carmen Paradas , Tanya Stojkovic , Madoka Mori-Yoshimura , Elena Bravver , Jordi Díaz-Manera , Elena Pegoraro , Jerry R Mendell , Auteur inconnu , Anna G Mayhew , Volker Straub

Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy.

Neurology
Avec: Diane E Frank , Frederick J Schnell , Cody Akana , Saleh H El-Husayni , Cody A Desjardins , Jennifer Morgan , Jay S Charleston , Valentina Sardone , Joana Domingos , George Dickson , Volker Straub , Michela Guglieri , Eugenio Mercuri , Laurent Servais , Francesco Muntoni , Auteur inconnu

Observational study of clinical outcomes for testosterone treatment of pubertal delay in Duchenne muscular dystrophy.

BMC pediatrics
Avec: C L Wood , T D Cheetham , K G Hollingsworth , M Guglieri , Y Ailins-Sahun , S Punniyakodi , A Mayhew , V Straub

Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study.

PLoS medicine
Avec: Edward C Smith , Laurie S Conklin , Eric P Hoffman , Paula R Clemens , Jean K Mah , Richard S Finkel , Michela Guglieri , Mar Tulinius , Yoram Nevo , Monique M Ryan , Richard Webster , Diana Castro , Nancy L Kuntz , Laurie Kerchner , Lauren P Morgenroth , Adrienne Arrieta , Maya Shimony , Mark Jaros , Phil Shale , Heather Gordish-Dressman , Laura Hagerty , Utkarsh J Dang , Jesse M Damsker , Benjamin D Schwartz , Laurel J Mengle-Gaw , Craig M McDonald , Auteur inconnu

Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy: a phase 2 trial of domagrozumab.

Journal of neurology
Avec: Sarah P Sherlock , Jeffrey Palmer , Kathryn R Wagner , Hoda Z Abdel-Hamid , Enrico Bertini , Cuixia Tian , Jean K Mah , Anna Kostera-Pruszczyk , Francesco Muntoni , Michela Guglieri , John F Brandsema , Eugenio Mercuri , Russell J Butterfield , Craig M McDonald , Lawrence Charnas , Shannon Marraffino

Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.

JAMA
Avec: Michela Guglieri , Kate Bushby , Michael P McDermott , Kimberly A Hart , Rabi Tawil , William B Martens , Barbara E Herr , Elaine McColl , Chris Speed , Jennifer Wilkinson , Janbernd Kirschner , Wendy M King , Michelle Eagle , Mary W Brown , Tracey Willis , Robert C Griggs , Auteur inconnu , Volker Straub , Henriette van Ruiten , Anne-Marie Childs , Emma Ciafaloni , Perry B Shieh , Stefan Spinty , Lorenzo Maggi , Giovanni Baranello , Russell J Butterfield , I A Horrocks , Helen Roper , Zoya Alhaswani , Kevin M Flanigan , Nancy L Kuntz , Adnan Manzur , Basil T Darras , Peter B Kang , Leslie Morrison , Monika Krzesniak-Swinarska , Jean K Mah , Tiziana E Mongini , Federica Ricci , Maja von der Hagen , Richard S Finkel , Kathleen O'Reardon , Matthew Wicklund , Ashutosh Kumar , Craig M McDonald , Jay J Han , Nanette Joyce , Erik K Henricson , Ulrike Schara-Schmidt , Andrea Gangfuss , Ekkehard Wilichowski , Richard J Barohn , Jeffrey M Statland , Craig Campbell , Giuseppe Vita , Gian Luca Vita , James F Howard , Imelda Hughes , Hugh J McMillan , Elena Pegoraro , Luca Bello , W Bryan Burnette , Mathula Thangarajh , Taeun Chang

Mitral transcatheter edge-to-edge repair vs. isolated mitral surgery for severe mitral regurgitation: a French nationwide study.

European heart journal
Avec: Pierre Deharo , Jean Francois Obadia , Patrice Guerin , Thomas Cuisset , Jean Francois Avierinos , Gilbert Habib , Olivier Torras , Arnaud Bisson , Pascal Vigny , Christophe Saint Etienne , Carl Semaan , Mickael Guglieri , Nicolas Dumonteil , Frederic Collart , Martine Gilard , Thomas Modine , Erwan Donal , Bernard Iung , Laurent Fauchier

Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial.

PloS one
Avec: Francesco Muntoni , Michela Guglieri , Jean K Mah , Kathryn R Wagner , John F Brandsema , Russell J Butterfield , Craig M McDonald , Anna G Mayhew , Jeffrey P Palmer , Shannon Marraffino , Lawrence Charnas , Eugenio Mercuri

Dual-energy X-ray absorptiometry measures of lean body mass as a biomarker for progression in boys with Duchenne muscular dystrophy.

Scientific reports
Avec: Sarah P Sherlock , Jeffrey Palmer , Kathryn R Wagner , Hoda Z Abdel-Hamid , Cuixia Tian , Jean K Mah , Francesco Muntoni , Michela Guglieri , Russell J Butterfield , Lawrence Charnas , Shannon Marraffino

Myostatin and follistatin as monitoring and prognostic biomarkers in dysferlinopathy.

Neuromuscular disorders : NMD
Avec: Ursula Moore , Esther Fernández-Simón , Marianela Schiava , Dan Cox , Heather Gordish-Dressman , Meredith K James , Anna Mayhew , Ian Wilson , Michela Guglieri , Laura Rufibach , Andrew Blamire , Pierre G Carlier , Madoka Mori-Yoshimura , John W Day , Kristi J Jones , Diana X Bharucha-Goebel , Emmanuelle Salort-Campana , Alan Pestronk , Maggie C Walter , Carmen Paradas , Tanya Stojkovic , Elena Bravver , Elena Pegoraro , Jerry R Mendell , Kate Bushby , Jordi Diaz-Manera , Volker Straub , Auteur inconnu

Analysis of muscle magnetic resonance imaging of a large cohort of patient with VCP-mediated disease reveals characteristic features useful for diagnosis.

Journal of neurology
Avec: Diana Esteller , Marianela Schiava , Rocío-Nur Villar-Quiles , Boris Dibowski , Nadia Venturelli , Pascal Laforet , Jorge Alonso-Pérez , Montse Olive , Cristina Domínguez-González , Carmen Paradas , Beatriz Vélez , Anna Kostera-Pruszczyk , Biruta Kierdaszuk , Carmelo Rodolico , Kristl Claeys , Endre Pál , Edoardo Malfatti , Sarah Souvannanorath , Alicia Alonso-Jiménez , Willem de Ridder , Eline De Smet , George Papadimas , Constantinos Papadopoulos , Sofia Xirou , Sushan Luo , Nuria Muelas , Juan J Vilchez , Alba Ramos-Fransi , Mauro Monforte , Giorgio Tasca , Bjarne Udd , Johanna Palmio , Srtuhi Sri , Sabine Krause , Benedikt Schöser , Roberto Fernández-Torrón , Adolfo López de Munain , Elena Pegoraro , Maria Elena Farrugia , Mathias Vorgerd , Georgious Manousakis , Jean Baptiste Chanson , Aleksandra Nadaj-Pakleza , Hakan Cetin , Umesh Badrising , Jodi Warman-Chardon , Jorge Bevilacqua , Nicholas Earle , Mario Campero , Jorge Díaz , Chiseko Ikenaga , Thomas E Lloyd , Ichizo Nishino , Yukako Nishimori , Yoshihiko Saito , Yasushi Oya , Yoshiaki Takahashi , Atsuko Nishikawa , Ryo Sasaki , Chiara Marini-Bettolo , Michela Guglieri , Volker Straub , Tanya Stojkovic , Robert Y Carlier , Jordi Díaz-Manera

Clinical Classification of Variants in the Valosin-Containing Protein Gene Associated With Multisystem Proteinopathy.

Neurology. Genetics
Avec: Marianela Schiava , Chiseko Ikenaga , Ana Topf , Marta Caballero-Ávila , Tsui-Fen Chou , Shan Li , Feng Wang , Jil Daw , Tanya Stojkovic , Rocio Villar-Quiles , Ichizo Nishino , Michio Inoue , Yukako Nishimori , Yoshihiko Saito , Masahisa Katsuno , Seiya Noda , Chihiro Ito , Mieko Otsuka , Sruthi Nahir , Georgios Manousakis , David Walk , Colin Quinn , Lindsay Alfano , Zarife Sahenk , Giorgio Tasca , Mauro Monforte , Mario Sabatelli , Giulia Bisogni , Anders Oldfors , Anna Rydeliu , Endre Pal , Carmen Paradas , Beatriz Velez , Jan L De Bleecker , Maria Elena Farugia , Cheryl Longman , Matthew B Harms , Stuart Ralston , Edmar Zanoteli , Andre Macedo Serafim da Silva , Javier Sotoca , Raul Juntas-Morales , Jorge Bevilacqua , Mireya Balart , Stuart Talbot , Volker Straub , Michela Guglieri , Chiara Marini-Bettolo , Jordi Diaz-Manera , Conrad Chris Weihl

An update on Becker muscular dystrophy.

Current opinion in neurology
Avec: Volker Straub , Michela Guglieri

Decoding the transcriptome of Duchenne muscular dystrophy to the single nuclei level reveals clinical-genetic correlations.

Cell death & disease
Avec: Xavier Suárez-Calvet , Esther Fernández-Simón , Daniel Natera , Cristina Jou , Patricia Pinol-Jurado , Elisa Villalobos , Carlos Ortez , Alexandra Monceau , Marianela Schiava , Anna Codina , José Verdu-Díaz , James Clark , Zoe Laidler , Priyanka Mehra , Rasya Gokul-Nath , Jorge Alonso-Perez , Chiara Marini-Bettolo , Giorgio Tasca , Volker Straub , Michela Guglieri , Andrés Nascimento , Jordi Diaz-Manera

Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy.

Neuromuscular disorders : NMD
Avec: Kathryn R Wagner , Hoda Z Abdel-Hamid , Jean K Mah , Craig Campbell , Michela Guglieri , Francesco Muntoni , Yasuhiro Takeshima , Craig M McDonald , Anna Kostera-Pruszczyk , Peter Karachunski , Russell J Butterfield , Eugenio Mercuri , Chiara Fiorillo , Enrico S Bertini , Cuixia Tian , Jeffery Statland , Alesia B Sadosky , Vivek S Purohit , Sarah P Sherlock , Jeffrey P Palmer , Michael Binks , Lawrence Charnas , Shannon Marraffino , Brenda L Wong

The CINRG Becker Natural History Study: Baseline characteristics.

Muscle & nerve
Avec: Paula R Clemens , Gabriela Niizawa , Jia Feng , Julaine Florence , Andrea Smith DʼAlessandro , Lauren P Morgenroth , Ksenija Gorni , Michela Guglieri , Anne Connolly , Matthew Wicklund , Tulio Bertorini , Jean K Mah , Mathula Thangarajh , Edward Smith , Nancy Kuntz , Craig M McDonald , Erik K Henricson , Saila Upadhyayula , Barry Byrne , Georgios Manousakis , Amy Harper , Elena Bravver , Susan Iannaccone , Christopher Spurney , Avital Cnaan , Heather Gordish-Dressman , Auteur inconnu

Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function.

Neurology
Avec: Eric P Hoffman , Benjamin D Schwartz , Laurel J Mengle-Gaw , Edward C Smith , Diana Castro , Jean K Mah , Craig M McDonald , Nancy L Kuntz , Richard S Finkel , Michela Guglieri , Katharine Bushby , Mar Tulinius , Yoram Nevo , Monique M Ryan , Richard Webster , Andrea L Smith , Lauren P Morgenroth , Adrienne Arrieta , Maya Shimony , Catherine Siener , Mark Jaros , Phil Shale , John M McCall , Kanneboyina Nagaraju , John van den Anker , Laurie S Conklin , Avital Cnaan , Heather Gordish-Dressman , Jesse M Damsker , Paula R Clemens , Auteur inconnu

Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy.

PloS one
Avec: Francesco Muntoni , Joana Domingos , Adnan Y Manzur , Anna Mayhew , Michela Guglieri , Auteur inconnu , Gautam Sajeev , James Signorovitch , Susan J Ward

Life Expectancy in Duchenne Muscular Dystrophy: Reproduced Individual Patient Data Meta-analysis.

Neurology
Avec: Jonathan Broomfield , Micki Hill , Michela Guglieri , Michael Crowther , Keith Abrams

Global Collaborative Social Network (Share4Rare) to Promote Citizen Science in Rare Disease Research: Platform Development Study.

JMIR formative research
Avec: Roxana Radu , Sara Hernández-Ortega , Oriol Borrega , Avril Palmeri , Dimitrios Athanasiou , Nicholas Brooke , Inma Chapí , Anaïs Le Corvec , Michela Guglieri , Alexandre Perera-Lluna , Jon Garrido-Aguirre , Bettina Ryll , Begonya Nafria Escalera

Cardiac and pulmonary findings in dysferlinopathy: A 3-year, longitudinal study.

Muscle & nerve
Avec: Ursula Moore , Roberto Fernandez-Torron , Marni Jacobs , Heather Gordish-Dressman , Jordi Diaz-Manera , Meredith K James , Anna G Mayhew , Elizabeth Harris , Michela Guglieri , Laura E Rufibach , Jia Feng , Andrew M Blamire , Pierre G Carlier , Simone Spuler , John W Day , Kristi J Jones , Diana X Bharucha-Goebel , Emmanuelle Salort-Campana , Alan Pestronk , Maggie C Walter , Carmen Paradas , Tanya Stojkovic , Madoka Mori-Yoshimura , Elena Bravver , Elena Pegoraro , Linda Pax Lowes , Jerry R Mendell , Kate Bushby , Auteur inconnu , John Bourke , Volker Straub

Muscle magnetic resonance imaging of a large cohort of distal hereditary motor neuropathies reveals characteristic features useful for diagnosis.

Neuromuscular disorders : NMD
Avec: Diana Esteller , Jasper Morrow , Jorge Alonso-Pérez , David Reyes , Alvaro Carbayo , Giulia Bisogni , Michela Cateruccia , Mauro Monforte , Giorgio Tasca , Aljwhara Alangary , Chiara Marini-Bettolo , Mario Sabatelli , Matilde Laura , Gita Ramdharry , Carla Bolaño-Díaz , Janina Turon-Sans , Ana Töpf , Michella Guglieri , Alexander M Rossor , Montse Olive , Enrico Bertini , Volker Straub , Mary M Reilly , Ricard Rojas-García , Jordi Díaz-Manera

Developing a Natural History Model for Duchenne Muscular Dystrophy.

PharmacoEconomics - open
Avec: Jonathan Broomfield , M Hill , F Chandler , M J Crowther , J Godfrey , M Guglieri , J Hastie , J Larkindale , J Mumby-Croft , E Reuben , F Woodcock , K R Abrams , Auteur inconnu

Findings from the Longitudinal CINRG Becker Natural History Study.

Journal of neuromuscular diseases
Avec: Paula R Clemens , Heather Gordish-Dressman , Gabriela Niizawa , Ksenija Gorni , Michela Guglieri , Anne M Connolly , Matthew Wicklund , Tulio Bertorini , Jean Mah , Mathula Thangarajh , Edward C Smith , Nancy L Kuntz , Craig M McDonald , Erik Henricson , S Upadhyayula , Barry Byrne , Georgios Manousakis , Amy Harper , Susan Iannaccone , Utkarsh J Dang

Identification of a novel heterozygous DYSF variant in a large family with a dominantly-inherited dysferlinopathy.

Neuropathology and applied neurobiology
Avec: Chiara Folland , Russell Johnsen , Adriana Botero Gomez , Daniel Trajanoski , Mark R Davis , Ursula Moore , Volker Straub , Rita Barresi , Michela Guglieri , Hannah Hayhurst , Andrew M Schaefer , Nigel G Laing , Philipa J Lamont , Gianina Ravenscroft

Cardiac care of children with dystrophinopathy and females carrying DMD-gene variations.

Open heart
Avec: John Bourke , Cathy Turner , William Bradlow , Ashish Chikermane , Caroline Coats , Matthew Fenton , Maria Ilina , Alexandra Johnson , Stam Kapetanakis , Lisa Kuhwald , Adrian Morley-Davies , Ros Quinlivan , Konstantinos Savvatis , Marianela Schiava , Zaheer Yousef , Michela Guglieri

Functional abilities, respiratory and cardiac function in a large cohort of adults with Duchenne muscular dystrophy treated with glucocorticoids.

European journal of neurology
Avec: Marianela Schiava , Robert Muni Lofra , John P Bourke , Jordi Díaz-Manera , Meredith K James , Maha A Elseed , Monika Malinova , Jassi Michel-Sodhi , Dionne Moat , Elisabetta Ghimenton , Michelle Mccallum , Carla Florencia Bolaño Díaz , Anna Mayhew , Karen Wong , Mark Richardson , Giorgio Tasca , Gail Eglon , Michelle Eagle , Cathy Turner , Emma Heslop , Volker Straub , Chiara Marini Bettolo , Michela Guglieri

Imaging mass cytometry analysis of Becker muscular dystrophy muscle samples reveals different stages of muscle degeneration.

Scientific reports
Avec: Patricia Piñol-Jurado , José Verdú-Díaz , Esther Fernández-Simón , Cristina Domínguez-González , Aurelio Hernández-Lain , Conor Lawless , Amy Vincent , Alejandro González-Chamorro , Elisa Villalobos , Alexandra Monceau , Zoe Laidler , Priyanka Mehra , James Clark , Andrew Filby , David McDonald , Paul Rushton , Andrew Bowey , Jorge Alonso Pérez , Giorgio Tasca , Chiara Marini-Bettolo , Michela Guglieri , Volker Straub , Xavier Suárez-Calvet , Jordi Díaz-Manera

Disease-associated comorbidities, medication records and anthropometric measures in adults with Duchenne muscular dystrophy.

Neuromuscular disorders : NMD
Avec: Marianela Schiava , Robert Muni Lofra , John P Bourke , Meredith K James , Jordi Díaz-Manera , Maha A Elseed , Jassi Michel-Sodhi , Dionne Moat , Michelle Mccallum , Anna Mayhew , Elisabetta Ghimenton , Carla Florencia Bolaño Díaz , Monika Malinova , Karen Wong , Mark Richardson , Giorgio Tasca , Emma Grover , Emma-Jayne Robinson , Stephanie Tanner , Gail Eglon , Laura Behar , Michelle Eagle , Catherine Turner , José Verdú-Díaz , Emma Heslop , Volker Straub , Chiara Marini Bettolo , Michela Guglieri

Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial.

Neurology
Avec: Utkarsh J Dang , Jesse M Damsker , Michela Guglieri , Paula R Clemens , Seth J Perlman , Edward C Smith , Iain Horrocks , Richard S Finkel , Jean K Mah , Nicolas Deconinck , Nathalie M Goemans , Jana Haberlová , Volker Straub , Laurel Mengle-Gaw , Benjamin D Schwartz , Amy Harper , Perry B Shieh , Liesbeth De Waele , Diana Castro , Michele L Yang , Monique M Ryan , Craig M McDonald , Mar Tulinius , Richard I Webster , Hugh J Mcmillan , Nancy Kuntz , Vamshi K Rao , Giovanni Baranello , Stefan Spinty , Anne-Marie Childs , Annie M Sbrocchi , Kathryn A Selby , Migvis Monduy , Yoram Nevo , Juan J Vilchez , Andres Nascimento-Osorio , Erik H Niks , Imelda J M De Groot , Marina Katsalouli , John N Van Den Anker , Leanne M Ward , Mika Leinonen , Andrea L D'Alessandro , Eric P Hoffman

Reductions in functional muscle mass and ability to ambulate in Duchenne muscular dystrophy from ages 4 to 24 years.

The Journal of physiology
Avec: William J Evans , Marc Hellerstein , Russell J Butterfield , Edward Smith , Michela Guglieri , Natalie Katz , Brittany Nave , Lauren Branigan , Stephanie Thera , Kalista L Vordos , Laura Behar , Marianela Schiava , Meredith K James , Tyler Field , Hussein Mohammed , Mahalakshmi Shankaran

Réseau de co-auteurs

Michela Guglieri 60 collaborations
Volker Straub 30 collaborations
None None 21 collaborations
Craig M McDonald 13 collaborations
Marianela Schiava 13 collaborations
Francesco Muntoni 12 collaborations
Jean K Mah 12 collaborations
Meredith K James 10 collaborations
Jordi Díaz-Manera 10 collaborations
Anna Mayhew 9 collaborations
Heather Gordish-Dressman 9 collaborations
Paula R Clemens 9 collaborations
Elena Pegoraro 9 collaborations
Giorgio Tasca 9 collaborations
Chiara Marini-Bettolo 8 collaborations
Ursula Moore 8 collaborations
Carmen Paradas 8 collaborations
Kate Bushby 7 collaborations
Edward C Smith 7 collaborations
Michael P McDermott 7 collaborations
Robert C Griggs 7 collaborations
Utkarsh J Dang 7 collaborations
Eric P Hoffman 7 collaborations
Eugenio Mercuri 7 collaborations
Anna G Mayhew 7 collaborations
Jordi Diaz-Manera 7 collaborations
Tanya Stojkovic 7 collaborations
Monique M Ryan 6 collaborations
Mar Tulinius 6 collaborations
Richard S Finkel 6 collaborations
Nancy L Kuntz 6 collaborations
Luca Bello 6 collaborations
Elena Bravver 6 collaborations
Russell J Butterfield 6 collaborations
Mathula Thangarajh 5 collaborations
Richard Webster 5 collaborations
Benjamin D Schwartz 5 collaborations
Diana Castro 5 collaborations
Yoram Nevo 5 collaborations
Jesse M Damsker 5 collaborations
Laurent Servais 5 collaborations
Michelle Eagle 5 collaborations
Jia Feng 5 collaborations
Pierre G Carlier 5 collaborations
John W Day 5 collaborations
Kristi J Jones 5 collaborations
Diana X Bharucha-Goebel 5 collaborations
Emmanuelle Salort-Campana 5 collaborations
Alan Pestronk 5 collaborations
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Madoka Mori-Yoshimura 5 collaborations
Jerry R Mendell 5 collaborations
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Stefan Spinty 4 collaborations
Elaine McColl 4 collaborations
Robert Muni-Lofra 4 collaborations
Amy Harper 4 collaborations
William B Martens 4 collaborations
Laurel J Mengle-Gaw 4 collaborations
Giovanni Baranello 4 collaborations
Anne-Marie Childs 4 collaborations
Erik H Niks 4 collaborations
Leanne M Ward 4 collaborations
Jonathan Broomfield 4 collaborations
Cuixia Tian 4 collaborations
M Guglieri 4 collaborations
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