Co-existence of juvenile-onset systemic lupus erythematosus and juvenile myasthenia gravis
Sampath, S, McCann, L J, McDonagh, J, Cleary, G, Spinty, S, Pain, C E, Baildam, E M, Beresford, M W
Published in Lupus (01.11.2015)
Published in Lupus (01.11.2015)
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Journal Article
Overcoming pharmacokinetic challenges to drug administration in Duchenne muscular dystrophy: Lessons from Phase 1 development of ezutromid
Tejura, B, Spinty, S, Roper, H, Hughes, I, Majumdar, A, Harriman, S, Davies, K, Muntoni, F
Published in European journal of paediatric neurology (01.06.2017)
Published in European journal of paediatric neurology (01.06.2017)
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Journal Article
Utrophin modulators to treat Duchenne muscular dystrophy (DMD): Results from a Phase 1b Clinical Trial of SMT C1100
Tinsley, J, Muntoni, F, Spinty, S, Roper, H, Hughes, I, Ricotti, R, Tejura, B, Layton, G, Davies, K
Published in Neuromuscular disorders : NMD (01.10.2015)
Published in Neuromuscular disorders : NMD (01.10.2015)
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Spontaneous spinal epidural haematoma mimicking meningitis in a 2-year-old child—a case report and literature review
Jumani, D. B., Littlewood, R., Iyer, A., Fellows, G., Healey, A., Abernethy, L., Spinty, S., Sarginson, R., Pettorini, B.
Published in Child's nervous system (01.10.2013)
Published in Child's nervous system (01.10.2013)
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Journal Article
P06 Audit of unexpected weight loss in patients with Duchenne muscular dystrophy
Kulshrestha, R, Swiderska, N, Stuart, H, Spinty, S
Published in Neuromuscular disorders : NMD (01.03.2012)
Published in Neuromuscular disorders : NMD (01.03.2012)
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Journal Article
G.P.102
Muntoni, F, Spinty, S, Roper, H, Hughes, I, Ricotti, V, Bracchi, A, Horne, G, Tinsley, J
Published in Neuromuscular disorders : NMD (01.10.2014)
Published in Neuromuscular disorders : NMD (01.10.2014)
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Journal Article
Novel compound heterozygous mutations in the desmoplakin gene cause hair shaft abnormalities and culminate in lethal cardiomyopathy
Yesudian, P. D., Cabral, R. M., Ladusans, E., Spinty, S., Gibbs, J., Fryer, A., Christiano, A. M., Mendelsohn, S. S.
Published in Clinical and experimental dermatology (01.06.2014)
Published in Clinical and experimental dermatology (01.06.2014)
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P139 Intravenous levetiracetam: a potential new treatment option for acute seizures
McTague, A, Appleton, R, Kneen, R, Spinty, S, Curran, A, Kumar, R
Published in European journal of paediatric neurology (2009)
Published in European journal of paediatric neurology (2009)
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Journal Article
Second case of a successful pregnancy in maternal isovaleric acidaemia
SPINTY, S, ROGOZINSKI, H, LEALMAN, G. T, WRAITH, J. E
Published in Journal of inherited metabolic disease (01.12.2002)
Published in Journal of inherited metabolic disease (01.12.2002)
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Journal Article
The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials
Ricotti, Valeria, Ridout, Deborah A, Pane, Marika, Main, Marion, Mayhew, Anna, Mercuri, Eugenio, Manzur, Adnan Y, Muntoni, Francesco
Published in Journal of neurology, neurosurgery and psychiatry (01.02.2016)
Published in Journal of neurology, neurosurgery and psychiatry (01.02.2016)
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Journal Article
Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy
Muntoni, Francesco, Domingos, Joana, Manzur, Adnan Y, Mayhew, Anna, Guglieri, Michela, Sajeev, Gautam, Signorovitch, James, Ward, Susan J
Published in PloS one (03.09.2019)
Published in PloS one (03.09.2019)
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Journal Article
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
Joseph, Shuko, Wang, Cunyi, Bushby, Kate, Guglieri, Michaela, Horrocks, Iain, Straub, Volker, Ahmed, S Faisal, Wong, Sze Choong
Published in Archives of neurology (Chicago) (01.06.2019)
Published in Archives of neurology (Chicago) (01.06.2019)
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Journal Article
Long-term benefits and adverse effects of intermittent versus daily glucocorticoids in boys with Duchenne muscular dystrophy
Ricotti, Valeria, Ridout, Deborah A, Scott, Elaine, Quinlivan, Ros, Robb, Stephanie A, Manzur, Adnan Y, Muntoni, Francesco
Published in Journal of neurology, neurosurgery and psychiatry (01.06.2013)
Published in Journal of neurology, neurosurgery and psychiatry (01.06.2013)
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Journal Article
P.133 Daily regimens of prednisone, deflazacort and vamorolone improve motor function similarly in patients with Duchenne muscular dystrophy
McDonald, C., Henricson, E., Leinonen, M., Linden, A., Guglieri, M., Clemens, P., Griggs, R., Shieh, P., Horrocks, S., Mah, J., Finkel, R., Goemans, N., Straub, V., Ryan, M., McMillan, H., Spinty, S., Hoffman, E.
Published in Neuromuscular disorders : NMD (01.10.2022)
Published in Neuromuscular disorders : NMD (01.10.2022)
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Journal Article
FP.27 Results of a double-blind cross-over trial of vamorolone in DMD: A safer alternative to corticosteroids
Hoffman, E., Guglieri, M., Clemens, P., Perlman, S., Smith, E., Horrocks, I., Finkel, R., Mah, J., Deconinck, N., Goemans, N., Haberlova, J., Straub, V., Harper, A., Webster, R., McMillan, H., Baranello, G., Spinty, S., Childs, A., Selby, K., Vilchez-Padilla, J., Niks, E.
Published in Neuromuscular disorders : NMD (01.10.2022)
Published in Neuromuscular disorders : NMD (01.10.2022)
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Journal Article
Development of a consensus core dataset in juvenile dermatomyositis for clinical use to inform research
McCann, Liza J, Pilkington, Clarissa A, Huber, Adam M, Ravelli, Angelo, Appelbe, Duncan, Kirkham, Jamie J, Williamson, Paula R, Aggarwal, Amita, Christopher-Stine, Lisa, Constantin, Tamas, Feldman, Brian M, Lundberg, Ingrid, Maillard, Sue, Mathiesen, Pernille, Murphy, Ruth, Pachman, Lauren M, Reed, Ann M, Rider, Lisa G, van Royen-Kerkof, Annet, Russo, Ricardo, Spinty, Stefan, Wedderburn, Lucy R, Beresford, Michael W
Published in Annals of the rheumatic diseases (01.02.2018)
Published in Annals of the rheumatic diseases (01.02.2018)
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