Professor Michela Guglieri
| Assessing Pharmacogenomic loci Associated with the Pharmacokinetics of Vamorolone in Boys with Duchenne Muscular Dystrophy | 2024 |
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Dr Doaa Salman Carla Bolaño Diaz Robert Muni Lofra Karen Wong Dr Maha Elseed et al. | Axial involvement as a prominent feature in SMPX-related distal myopathy | 2024 |
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Vicky Mercer Dr Sharmila Jandial Professor Michela Guglieri Emerita Professor Helen Foster
| Beyond pGALS: the need for a multifaceted musculoskeletal decision-making tool ('pGALSplus') in community-based clinical practice | 2024 |
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Professor Michela Guglieri
| Developing a Natural History Model for Duchenne Muscular Dystropgy | 2024 |
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Catherine Turner Dr John Bourke Meredith James Jassi Sodhi Dr Marianela Schiava et al. | Development of respiratory care guidelines for Duchenne muscular dystrophy in the UK: key recommendations for clinical practice | 2024 |
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Dr Marianela Schiava Robert Muni Lofra Dr John Bourke Meredith James Professor Jordi Diaz Manera et al. | Disease-associated comorbidities, medication records and anthropometric measures in adults with Duchenne muscular dystrophy | 2024 |
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Professor Michela Guglieri Dr Jana Haberlova Professor Volker Straub Dr Liesbeth De Waele
| Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy; A Randomized Controlled Trial | 2024 |
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Dr Marianela Schiava Dr Anna Mayhew Professor Michela Guglieri
| Factors Associated With Early Motor Function Trajectories in DMD After Glucocorticoid Initiation: Post Hoc Analysis of the FOR-DMD Trial | 2024 |
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Professor Michela Guglieri
| Findings from the Longitudinal CINRG Becker Natural History Study | 2024 |
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Dr Marianela Schiava Dr John Bourke Meredith James Dr Maha Elseed Dr Monika Malinova et al. | Functional abilities, respiratory and cardiac function in a large cohort of adults with Duchenne muscular dystrophy treated with glucocorticoids | 2024 |
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Patricia Pinol Jurado Jose Verdú-Díaz Esther Fernández-Simón Dr Conor Lawless Dr Amy Vincent et al. | Imaging mass cytometry analysis of Becker muscular dystrophy muscle samples reveals different stages of muscle degeneration | 2024 |
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Catherine Turner Professor Michela Guglieri
| Moving Beyond the 2018 Minimum International Care Considerations for Osteoporosis Management in Duchenne Muscular Dystrophy (DMD): Meeting Report from the 3rd International Muscle-Bone Interactions Meeting 7th and 14th November 2022 | 2024 |
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Professor Michela Guglieri Laura Behar Dr Marianela Schiava Meredith James
| Reductions in functional muscle mass and ability to ambulate in Duchenne muscular dystrophy from ages 4 to 24 years | 2024 |
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Dr Marianela Schiava Dr Anna Mayhew Professor Michela Guglieri
| Reference curves of motor function outcomes in young steroid-naïve males with Duchenne muscular dystrophy | 2024 |
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Vicky Mercer Professor Michela Guglieri Professor Jeremy Parr Emerita Professor Helen Foster Dr Sharmila Jandial et al. | The development of pGALSplus: evaluating feasibility and acceptability of an assessment to facilitate the identification and triage of children with musculoskeletal presentations | 2024 |
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Dr Marianela Schiava Jose Verdú-Díaz Professor Giorgio Tasca Professor Volker Straub Professor Chiara Marini Bettolo et al. | Analysis of muscle magnetic resonance imaging of a large cohort of patient with VCP-mediated disease reveals characteristic features useful for diagnosis | 2023 |
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Esther Fernández-Simón Patricia Pinol Jurado Dr Elisa Villalobos Villegas Dr Alexandra Monceau Dr Marianela Schiava et al. | Decoding the transcriptome of Duchenne muscular dystrophy to the single nuclei level reveals clinical-genetic correlations | 2023 |
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Professor Volker Straub Professor Michela Guglieri
| DMD Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical Trials | 2023 |
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Dr Claire Wood Dr Kieren Hollingsworth Edrina Bokaie Dr Eric Hughes Robert Muni Lofra et al. | Is ongoing testosterone required after pubertal induction in Duchenne muscular dystrophy? | 2023 |
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Professor Giorgio Tasca Professor Chiara Marini Bettolo Dr Ana Topf Professor Michela Guglieri Professor Volker Straub et al. | Muscle magnetic resonance imaging of a large cohort of distal hereditary motor neuropathies reveals characteristic features useful for diagnosis | 2023 |
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Dr Ursula Moore Esther Fernández-Simón Dr Marianela Schiava Daniel Cox Meredith James et al. | Myostatin and follistatin as monitoring and prognostic biomarkers in dysferlinopathy | 2023 |
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Professor Michela Guglieri
| Natural History of Duchenne muscular dystrophy in the United Kingdom: A descriptive study using the Clinical Practice Research Datalink | 2023 |
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Professor Volker Straub Professor Michela Guglieri
| T Cell Responses to Dystrophin in a Natural History Study of Duchenne Muscular Dystrophy | 2023 |
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Andrew Galloway Daniel Cox Dr Ana Topf Heather Hilsden Professor Michela Guglieri et al. | The John Walton Muscular Dystrophy Research Centre Biobank | 2023 |
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Meredith James Dr Michelle Eagle Robert Muni Lofra Dr ursula Moore Professor Michela Guglieri et al. | Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale | 2022 |
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Dr Anna Mayhew Meredith James Dr ursula Moore Helen Sutherland Robert Muni Lofra et al. | Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach | 2022 |
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Dr ursula Moore Roberto Fernandez-Torron Meredith James Dr Anna Mayhew Dr Lizzie Harris et al. | Cardiac and pulmonary findings in dysferlinopathy: A 3-year, longitudinal study | 2022 |
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Dr John Bourke Catherine Turner Dr Marianela Schiava Professor Michela Guglieri
| Cardiac care of children with dystrophinopathy and females carrying DMD-gene variations | 2022 |
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Dr Marianela Schiava Dr Henriette van Ruiten Dr Anna Mayhew Emerita Professor Elaine McColl Dr Tracey Willis et al. | Clinical and Genetic Characteristics in Young, Glucocorticoid-Naive Boys With Duchenne Muscular Dystrophy. | 2022 |
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Professor Michela Guglieri
| Dual-energy X-ray absorptiometry measures of lean body mass as a biomarker for progression in boys with Duchenne muscular dystrophy | 2022 |
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Dr Anna Mayhew Professor Michela Guglieri
| Editorial: Lessons Learned from Translational Research in Neuromuscular Diseases: Impact on Study Design, Outcome Measures and Managing Expectation | 2022 |
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Professor Michela Guglieri Emerita Professor Katherine Bushby Emerita Professor Elaine McColl Chris Speed Jennifer Wilkinson et al. | Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial | 2022 |
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Professor Michela Guglieri
| Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial | 2022 |
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Professor Michela Guglieri
| Efficacy and Safety of Vamorolone vs Placebo and Prednisone among Boys with Duchenne Muscular Dystrophy: A Randomized Clinical Trial | 2022 |
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Dr Anna Mayhew Dionne Moat Dr Michelle Eagle Meredith James Robert Muni Lofra et al. | Functional outcome measures in young, steroid-naïve boys with Duchenne muscular dystrophy | 2022 |
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Dr ursula Moore Professor Volker Straub Dr Rita Barresi Professor Michela Guglieri Dr Hannah Hayhurst et al. | Identification of a novel heterozygous DYSF variant in a large family with a dominantly-inherited dysferlinopathy | 2022 |
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Professor Volker Straub Professor Michela Guglieri
| 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 | 2022 |
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Professor Michela Guglieri Dr Anna Mayhew
| Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial | 2022 |
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Professor Michela Guglieri
| Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy: a phase 2 trial of domagrozumab | 2022 |
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Emma Heslop Catherine Turner Anna Irvin Professor Volker Straub Professor Michela Guglieri et al. | Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead | 2021 |
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Emerita Professor Elaine McColl Professor Michela Guglieri Professor Volker Straub
| Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy | 2021 |
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Dr Ursula Moore Meredith James Dr Anna Mayhew Professor Michela Guglieri Roberto Fernandez-Torron et al. | Miyoshi myopathy and limb girdle muscular dystrophy R2 are the same disease | 2021 |
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Dr John Bourke Gillian Watson Andrew Bryant Dr Thomas Chadwick Ruth Wood et al. | Preventing Cardiomyopathy in DMD | 2021 |
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Dr Claire Wood Dr Kieren Hollingsworth Dr Eric Hughes Dr Sadha Punniyakodi Robert Muni Lofra et al. | Pubertal induction in adolescents with DMD is associated with high satisfaction, gonadotropin release and increased muscle contractile surface area | 2021 |
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Professor Michela Guglieri Professor Volker Straub
| The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy | 2021 |
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Dr Claire Wood Dr Jonathon Foggin Professor Michela Guglieri Professor Volker Straub Professor Timothy Cheetham et al. | The impact of testosterone therapy on quality of life in adolescents with Duchenne muscular dystrophy | 2021 |
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Professor Michela Guglieri
| Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study | 2020 |
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Professor Michela Guglieri
| Multi-omics identifies circulating mirna and protein biomarkers for facioscapulohumeral dystrophy | 2020 |
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Professor Michela Guglieri Professor Volker Straub Dr Anna Sarkozy Dr Jana Haberlova Professor Giorgio Tasca et al. | New genotype-phenotype correlations in a large European cohort of patients with sarcoglycanopathy | 2020 |
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Professor Michela Guglieri
| Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy | 2020 |
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Professor Michela Guglieri
| The CINRG Becker Natural History Study: Baseline Characteristics | 2020 |
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Dr John Bourke Professor Michela Guglieri Professor Annemieke Aartsma-Rus Dr Guy MacGowan
| 238th ENMC International Workshop: Updating management recommendations of cardiac dystrophinopathyHoofddorp, The Netherlands, 30 November - 2 December 2018 | 2019 |
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Dr Anna Mayhew Professor Michela Guglieri
| Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy | 2019 |
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Emerita Professor Katherine Bushby Professor Michela Guglieri Professor Volker Straub
| 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 | 2019 |
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Dr Claire Wood Professor Timothy Cheetham Dr Kieren Hollingsworth Professor Michela Guglieri Dr Anna Mayhew et al. | Observational study of clinical outcomes for testosterone treatment of pubertal delay in Duchenne muscular dystrophy | 2019 |
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Dr Lizzie Harris Professor Chiara Marini Bettolo Dr Ana Topf Dr Rita Barresi Dr Tuomo Polvikoski et al. | MEGF10 related myopathies: A new case with adult onset disease with prominent respiratory failure and review of reported phenotypes | 2018 |
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Rebecca Crow Emerita Professor Elaine McColl Dr Jennifer Wilkinson Dr Michelle Eagle Professor Hanns Lochmuller et al. | A checklist for clinical trials in rare disease: obstacles and anticipatory actions—lessons learned from the FOR-DMD trial | 2018 |
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Professor Michela Guglieri
| A multinational study on motor function in early-onset FSHD | 2018 |
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Professor Michela Guglieri
| Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug | 2018 |
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Dr John Bourke Gillian Watson Professor Michela Guglieri Chris Speed Emerita Professor Elaine McColl et al. | Randomised placebo-controlled trial of combination ACE inhibitor and beta-blocker therapy to prevent cardiomyopathy in children with Duchenne muscular dystrophy? (DMD Heart Protection Study): a protocol study | 2018 |
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Professor Michela Guglieri Dr Alex Murphy Emerita Professor Katherine Bushby Dr John Bourke
| Cardiac involvement in female carriers of duchenne or becker muscular dystrophy | 2017 |
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Professor Michela Guglieri Emerita Professor Katherine Bushby Emerita Professor Elaine McColl Dr Jennifer Wilkinson Dr Michelle Eagle et al. | Developing standardized corticosteroid treatment for Duchenne muscular dystrophy | 2017 |
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Dr Cecilia Jimenez Moreno Cecilia Jimenez Moreno Dr Nikoletta Nikolenko Dr Jose Atalaia Professor Michela Guglieri et al. | Functional impairment in patients with myotonic dystrophy type 1 can be assessed by an ataxia rating scale (SARA) | 2017 |
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Dr Oksana Pogoryelova Professor Michela Guglieri Professor Chiara Marini Bettolo Professor Volker Straub Dr Teresinha Evangelista et al. | Reduced serum myostatin concentrations associated with genetic muscle disease progression | 2017 |
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Libby Wood Professor Chiara Marini Bettolo Professor Michela Guglieri Dr Grace McMacken Dr Anna Mayhew et al. | Respiratory involvement in ambulant and non-ambulant patients with facioscapulohumeral muscular dystrophy | 2017 |
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Dr Anna Mayhew Dr Michelle Eagle Professor Michela Guglieri Professor Volker Straub Professor Hanns Lochmuller et al. | Corrigendum to Development and psychometric analysis of the Duchenne muscular dystrophy Functional Ability Self-Assessment Tool (DMDSAT) (vol 25, pg 937, 2015) | 2016 |
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Libby Wood Roberto Fernandez-Torron Dr John Hudson Dr Fiona Norwood Professor Michela Guglieri et al. | Design, set-up and utility of the UK facioscapulohumeral muscular dystrophy patient registry | 2016 |
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Dr Andreas Roos Daniel Cox Dr Mojgan Reza Professor Michela Guglieri Professor Volker Straub et al. | MRC biobank Newcastle - A five-year review of the John Walton Muscular Dystrophy Research Centre experience | 2016 |
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Sebastian Figueroa Bonaparte Dr Rita Barresi Dr Tuomo Polvikoski Dr Timothy Williams Dr Ana Topf et al. | Mutational spectrum and phenotypic variability of VCP-related neurological disease in the UK | 2016 |
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Dr Michelle Eagle Dr Anna Mayhew Professor Michela Guglieri Professor Volker Straub
| Outcome measures for Duchenne muscular dystrophy from ambulant to non-ambulant: Implications for clinical trials | 2016 |
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Professor Michela Guglieri Dr Teresinha Evangelista Professor Volker Straub
| Prevalence of Pompe disease in 3,076 patients with hyperCKemia and limb-girdle muscular weakness | 2016 |
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David Rawlings Dr Claire Wood Professor Timothy Cheetham Dr Cecilia Jimenez Moreno Dr Anna Mayhew et al. | Prophylactic oral bisphosphonate therapy in duchenne muscular dystrophy | 2016 |
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Professor Michela Guglieri Professor Volker Straub Professor Hanns Lochmuller Emerita Professor Katherine Bushby
| Quantifying the burden of caregiving in Duchenne muscular dystrophy | 2016 |
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Dr Claire Wood Professor Volker Straub Professor Michela Guglieri Emerita Professor Katherine Bushby Professor Timothy Cheetham et al. | Short stature and pubertal delay in Duchenne muscular dystrophy | 2016 |
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Emerita Professor Katherine Bushby Professor Volker Straub Professor Michela Guglieri
| The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: Considerations for the design of clinical trials | 2016 |
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Dr Henriette van Ruiten Professor Chiara Marini Bettolo Professor Timothy Cheetham Dr Michelle Eagle Professor Hanns Lochmuller et al. | Why are some patients with Duchenne muscular dystrophy dying young: An analysis of causes of death in North East England | 2016 |
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Rebecca Crow Professor Michela Guglieri Emerita Professor Elaine McColl Dr Jennifer Wilkinson Dr Michelle Eagle et al. | Anticipating and overcoming obstacles in setting up NIH funded academic led, international clinical trials in rare disease - learning from for DMD | 2015 |
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Dr Anna Mayhew Dr Michelle Eagle Professor Michela Guglieri Professor Volker Straub Professor Hanns Lochmuller et al. | Development and Psychometric Analysis of the Duchenne Muscular Dystrophy Functional Ability Self-Assessment Tool (DMDSAT) | 2015 |
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Professor Michela Guglieri Professor Volker Straub Professor Giorgio Tasca
| Facilitating orphan drug development: Proceedings of the TREAT-NMD International Conference, December 2015, Washington, DC, USA | 2015 |
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Professor Michela Guglieri Professor Volker Straub Professor Hanns Lochmuller Emerita Professor Katherine Bushby
| Health-Related Quality of Life in Patients with Duchenne Muscular Dystrophy: A Multi-National, Cross-Sectional Study | 2015 |
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Dr Oksana Pogoryelova Professor Michela Guglieri Professor Volker Straub Emerita Professor Katherine Bushby Professor Hanns Lochmuller et al. | Muscle-Derived Proteins as Serum Biomarkers for Monitoring Disease Progression in Three Forms of Muscular Dystrophy | 2015 |
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Dr Michelle Eagle Professor Michela Guglieri Professor Volker Straub
| Outcome measures for Duchenne muscular dystrophy from ambulant to non-ambulant: Implications for clinical trials | 2015 |
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Dr Henriette van Ruiten Professor Michela Guglieri Dr Michelle Eagle Professor Volker Straub Professor Hanns Lochmuller et al. | Overview of morbidity, mortality and survival in Duchenne muscular dystrophy in the North East of England | 2015 |
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Professor Michela Guglieri Emerita Professor Elaine McColl Dr Jennifer Wilkinson Dr Michelle Eagle Emerita Professor Katherine Bushby et al. | Recruitment in the FOR-DMD study - double-blind randomized trial to optimize steroid regime in Duchenne Muscular Dystrophy (DMD) | 2015 |
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Dr Claire Wood Professor Timothy Cheetham Professor Michela Guglieri Emerita Professor Katherine Bushby Dr Catherine Owen et al. | Testosterone Treatment of Pubertal Delay in Duchenne Muscular Dystrophy | 2015 |
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Dr Alex Murphy Professor Michela Guglieri Emerita Professor Katherine Bushby Dr John Bourke
| The frequency and characterisation of cardiac involvement in female carriers of BMD or DMD: A cross sectional analysis | 2015 |
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Dr Richard Charlton Professor Michela Guglieri Emerita Professor Katherine Bushby Professor Volker Straub Dr Rita Barresi et al. | The importance of dosage analysis in dysferlinopathy | 2015 |
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Professor Michela Guglieri Dr Anna Sarkozy Dr Rita Barresi Professor Hanns Lochmuller Emerita Professor Katherine Bushby et al. | Autosomal recessive myofibrillar myopathy caused by ACTA1 mutations | 2014 |
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Professor Giorgio Tasca Professor Michela Guglieri Dr Rita Barresi Emerita Professor Katherine Bushby Professor Volker Straub et al. | Biochemical Characterization of Patients With In-Frame or Out-of-Frame DMD Deletions Pertinent to Exon 44 or 45 Skipping | 2014 |
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Dr Amina Chaouch Professor Michela Guglieri Professor Volker Straub Professor Hanns Lochmuller
| Fibronectin is a serum biomarker for Duchenne muscular dystrophy | 2014 |
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Dr Henriette van Ruiten Professor Volker Straub Emerita Professor Katherine Bushby Professor Michela Guglieri
| Improving recognition of Duchenne muscular dystrophy: a retrospective case note review | 2014 |
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Professor Michela Guglieri Dr Henriette van Ruiten Professor Volker Straub Emerita Professor Katherine Bushby
| Improving the diagnosis of Duchenne muscular dystrophy | 2014 |
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Dr Anna Sarkozy David Rawlings Professor Michela Guglieri Dr Kate Owen Professor Volker Straub et al. | Prophylactic oral bisphosphonate therapy in Duchenne muscular dystrophy: The Newcastle upon Tyne experience | 2014 |
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Professor Volker Straub Dr Michelle Eagle Professor Michela Guglieri
| Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study | 2014 |
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Professor Michela Guglieri Professor Volker Straub Professor Hanns Lochmuller Emerita Professor Katherine Bushby
| The burden of Duchenne muscular dystrophy: An international, cross-sectional study | 2014 |
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Dr Anna Sarkozy Dr Debbie Hicks Dr Steven Laval Dr Rita Barresi Dr Lizzie Harris et al. | ANO5 Gene Analysis in a Large Cohort of Patients with Anoctaminopathy: Confirmation of Male Prevalence and High Occurrence of the Common Exon 5 Gene Mutation | 2013 |
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Dr Anna Sarkozy Dr Debbie Hicks Dr Steven Laval Dr Rita Barresi Professor Michela Guglieri et al. | Clinical and molecular analysis of a large cohort of patients with anoctaminopathy | 2013 |
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Dr Mattia Calissano Professor Michela Guglieri Professor Volker Straub Emerita Professor Katherine Bushby
| Dystromirs as Serum Biomarkers for Monitoring the Disease Severity in Duchenne Muscular Dystrophy | 2013 |
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Emerita Professor Helen McConachie Professor Ann Le Couteur Professor Michela Guglieri Emerita Professor Katherine Bushby
| Emotional impact of genetic trials in progressive paediatric disorders: a dose-ranging exon-skipping trial in Duchenne muscular dystrophy | 2013 |
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Professor Giorgio Tasca Professor Michela Guglieri Dr Rita Barresi Emerita Professor Katherine Bushby Professor Volker Straub et al. | Biochemical and clinical variability of Becker muscular dystrophy: Predicting optimal target exons for exon skipping therapy in Duchenne muscular dystrophy | 2012 |
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Professor Michela Guglieri Dr Michelle Eagle Emerita Professor Katherine Bushby
| Body mass index (BMI) and growth in Duchenne Muscular Dystrophy (DMD) | 2011 |
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Professor Giorgio Tasca Professor Michela Guglieri Emerita Professor Katherine Bushby Professor Volker Straub
| Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials. | 2011 |
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Professor Michela Guglieri Dr John Bourke Professor Volker Straub Emerita Professor Katherine Bushby
| Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. | 2011 |
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Professor Michela Guglieri Dr Kieren Hollingsworth Ewan Mercer Professor Volker Straub Emerita Professor Katherine Bushby et al. | Muscle histology vs MRI in Duchenne muscular dystrophy | 2011 |
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Dr Mojgan Reza Professor Michela Guglieri Professor Hanns Lochmuller
| Two new protocols to enhance the production and isolation of human induced pluripotent stem cell lines | 2011 |
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Professor Michela Guglieri Emerita Professor Elaine McColl Dr Michelle Eagle Dr Karen Rafferty Emerita Professor Katherine Bushby et al. | FOR-DMD: double-blind randomized trial to optimize steroid regime in Duchenne Muscular Dystrophy (DMD) | 2010 |
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Professor Michela Guglieri Emerita Professor Katherine Bushby
| Molecular treatments in Duchenne muscular dystrophy | 2010 |
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Professor Volker Straub Emerita Professor Katherine Bushby Professor Michela Guglieri
| Recessive mutations in RYR1 are a common cause of congenital fiber type disproportion | 2010 |
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Professor Michela Guglieri Professor Volker Straub Emerita Professor Katherine Bushby
| Revertant fibres and dystrophin traces in Duchenne muscular dystrophy: Implication for clinical trials | 2010 |
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Professor Michela Guglieri Professor Volker Straub Emerita Professor Katherine Bushby
| Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study | 2009 |
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Professor Timothy Cheetham Professor Simon Pearce David Rawlings Dr Michelle Eagle Professor Michela Guglieri et al. | Oral bisphosphonates as prophylaxis of steroid-induced osteoporosis in Duchenne muscular dystrophy | 2009 |
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Professor Michela Guglieri Geoff Bell Dr John Bourke Dr Michelle Eagle Professor Patrick Chinnery et al. | Clinical follow up in a large cohort of patients affected by Myotonic Dystrophy type | 2008 |
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Professor Michela Guglieri Geoff Bell Dr John Bourke Dr Michelle Eagle Professor Patrick Chinnery et al. | D.P.2.02: Development of a nurse led multidisciplinary clinic for myotonic dystrophy type 1 | 2008 |
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Professor Michela Guglieri Geoff Bell Dr John Bourke Dr Michelle Eagle Professor Patrick Chinnery et al. | D.P.2.03: Clinical follow up in a large cohort of patients affected by myotonic dystrophy type 1 | 2008 |
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Professor Michela Guglieri Dr Michelle Eagle Dr John Bourke Professor Volker Straub Emerita Professor Katherine Bushby et al. | Delayed development and learning difficulties as a predominant symptom in female carriers of Duchenne and Becker muscular dystrophy | 2008 |
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Professor Michela Guglieri Geoff Bell Dr John Bourke Dr Michelle Eagle Professor Patrick Chinnery et al. | Development of a nurse led multidisciplinary clinic for myotonic dystrophy type 1 | 2008 |
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Professor Michela Guglieri Emerita Professor Katherine Bushby
| How to go about diagnosing and managing the limb-girdle muscular dystrophies | 2008 |
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Professor Michela Guglieri Dr Michelle Eagle Emerita Professor Katherine Bushby Professor Volker Straub
| Is assessment of respiratory function a good outcome measure for SMA II and III? | 2008 |
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Professor Michela Guglieri Professor Volker Straub Emerita Professor Katherine Bushby Professor Hanns Lochmuller
| Limb-girdle muscular dystrophies | 2008 |
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Dr Michelle Eagle Professor Michela Guglieri Professor Volker Straub Emerita Professor Katherine Bushby
| Long term steroid use in non-ambulatory patients with Duchenne muscular dystrophy | 2007 |
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Dr Michelle Eagle Professor Michela Guglieri Professor Volker Straub Emerita Professor Katherine Bushby
| Steroids in Duchenne muscular dystrophy (DMD): Natural history and clinical evaluation using the North Star Ambulatory Assessment (NSAA) | 2007 |
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