Search Results - "Manzur, Adnan"

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    Corticosteroids for the treatment of Duchenne muscular dystrophy by Matthews, Emma, Brassington, Ruth, Kuntzer, Thierry, Jichi, Fatima, Manzur, Adnan Y

    Published in Cochrane database of systematic reviews (05-05-2016)
    “…Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy of childhood. Untreated, this incurable disease, which has an X-linked recessive…”
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    Journal Article
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    Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy by 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)
    “…Functional variability among boys with Duchenne muscular dystrophy (DMD) is well recognised and complicates interpretation of clinical studies. We hypothesised…”
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    Journal Article
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    Peak functional ability and age at loss of ambulation in Duchenne muscular dystrophy by Zambon, Alberto A., Ayyar Gupta, Vandana, Ridout, Deborah, Manzur, Adnan Y., Baranello, Giovanni, Trucco, Federica, Muntoni, Francesco, Tirupath, Sandya, Douglas, Melanie, McFetridge, Jaci, Parasuraman, Deepak, Alhaswani, Zoya, McMurchie, Heather, Rabb, Rosanna, Majumdar, Anirban, Vijayakumar, Kayal, Amin, Sam, Mason, Faye, Frimpong‐Ansah, Claire, Gibbon, Frances, Parson, Bethan, Naismith, Karen, Burslem, Julie, Baxter, Alex, Eadie, Clare, Horrocks, Iain, Di Marco, Marina, Childs, Anne‐Marie, Pallant, Lindsey, Spinty, Stefan, Shillington, Alison, Gregson, Sarah, Cheshman, Laura, Wraige, Elizabeth, Gowda, Vasantha, Jungbluth, Heinz, Sheehan, Jennie, Hughes, Imelda, Warner, Sinead, Straub, Volker, Guglieri, Michela, Mayhew, Anna, Chow, Gabby, Williamson, Sarah, Willis, Tracey, Kulshrestha, Richa, Emery, Nicholas, Ramdas, Sithara, Ramjattan, Hayley, de Goede, Christian, Selley, Andrea, Ong, Min, White, Kay, Illingworth, Marjorie, Geary, Michelle, Palmer, Jenni, White, Cathy, Greenfield, Kate, Hewawitharana, Gemunu, Julien, Yvonne, Stephens, Elma, Tewnion, Jane, Ambegaonkar, Gautam, Krishnakumar, Deepa, Taylor, Jacqui, Ward, Catherine, Willis, Tracey, Wright, Elizabeth, Rylance, Claire

    Published in Developmental medicine and child neurology (01-08-2022)
    “…Aim To correlate the North Star Ambulatory Assessment (NSAA) and timed rise from floor (TRF) recorded at age of expected peak with age at loss of ambulation…”
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    Journal Article
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    Congenital myopathies: Natural history of a large pediatric cohort by Colombo, Irene, Scoto, Mariacristina, Manzur, Adnan Y, Robb, Stephanie A, Maggi, Lorenzo, Gowda, Vasantha, Cullup, Thomas, Yau, Michael, Phadke, Rahul, Sewry, Caroline, Jungbluth, Heinz, Muntoni, Francesco

    Published in Neurology (06-01-2015)
    “…OBJECTIVE:To assess the natural history of congenital myopathies (CMs) due to different genotypes. METHODS:Retrospective cross-sectional study based on…”
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    Journal Article
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    Cellular, biochemical and molecular changes in muscles from patients with X-linked myotubular myopathy due to MTM1 mutations by Bachmann, Christoph, Jungbluth, Heinz, Muntoni, Francesco, Manzur, Adnan Y, Zorzato, Francesco, Treves, Susan

    Published in Human molecular genetics (15-01-2017)
    “…Centronuclear myopathies are early-onset muscle diseases caused by mutations in several genes including MTM1, DNM2, BIN1, RYR1 and TTN. The most severe and…”
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    Journal Article
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    Respiratory Function and Sleep Disordered Breathing in Pediatric Duchenne Muscular Dystrophy by Zambon, Alberto A., Trucco, Federica, Laverty, Aidan, Riley, Mollie, Ridout, Deborah, Manzur, Adnan Y., Abel, Francois, Muntoni, Francesco

    Published in Neurology (20-09-2022)
    “…BACKGROUND AND OBJECTIVESThe decline of respiratory function in Duchenne muscular dystrophy (DMD) is associated with sleep disordered breathing (SDB) and…”
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    Skeletal Muscle Channelopathies: Rare Disorders with Common Pediatric Symptoms by Matthews, Emma, Silwal, Arpana, Sud, Richa, Hanna, Michael G., Manzur, Adnan Y., Muntoni, Francesco, Munot, Pinki

    Published in The Journal of pediatrics (01-09-2017)
    “…To ascertain the presenting symptoms of children with skeletal muscle channelopathies to promote early diagnosis and treatment. Retrospective case review of 38…”
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    The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials by Ricotti, Valeria, Ridout, Deborah A, Pane, Marika, Main, Marion, Mayhew, Anna, Mercuri, Eugenio, Manzur, Adnan Y, Muntoni, Francesco

    “…ObjectiveWith the emergence of experimental therapies for Duchenne muscular dystrophy (DMD), it is fundamental to understand the natural history of this…”
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    Long-term benefits and adverse effects of intermittent versus daily glucocorticoids in boys with Duchenne muscular dystrophy by Ricotti, Valeria, Ridout, Deborah A, Scott, Elaine, Quinlivan, Ros, Robb, Stephanie A, Manzur, Adnan Y, Muntoni, Francesco

    “…Objective To assess the current use of glucocorticoids (GCs) in Duchenne muscular dystrophy in the UK, and compare the benefits and the adverse events of daily…”
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    Journal Article
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