Search Results - "Guldberg, Christina"

Validation of the 5-domain Niemann-Pick type C Clinical Severity Scale by Patterson, Marc C, Lloyd-Price, Lucy, Guldberg, Christina, Doll, Helen, Burbridge, Claire, Chladek, Michael, íDali, Christine, Mengel, Eugen, Symonds, Tara

“…Niemann-Pick disease type C (NPC) is an ultra-rare, progressive, genetic disease leading to impaired lysosomal function and neurodegeneration causing serious…”
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International consensus on clinical severity scale use in evaluating Niemann-Pick disease Type C in paediatric and adult patients: results from a Delphi Study by Evans, William, Patterson, Marc, Platt, Frances, Guldberg, Christina, Mathieson, Toni, Pacey, Jessica

“…Several scales have been developed in the past two decades to evaluate Niemann-Pick disease Type C (NPC) severity in clinical practice and trials. However, a…”
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Impacts and Burden of Niemann pick Type-C: a patient and caregiver perspective by Mengel, Eugen, Patterson, Marc C, Chladek, Michael, Guldberg, Christina, Í Dali, Christine, Symonds, Tara, Lloyd-Price, Lucy, Mathieson, Toni, Crowe, Joslyn, Burbridge, Claire

“…Niemann-Pick disease type C (NPC) is a debilitating condition that impacts patients' and caregivers' quality of life (QOL) and reduces the patient's life…”
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Correction to: Clinical disease progression and biomarkers in Niemann–Pick disease type C: a prospective cohort study by Mengel, Eugen, Bembi, Bruno, del Toro, Mireia, Deodato, Federica, Gautschi, Matthias, Grunewald, Stephanie, Granborg, Sabine, Héron, Bénédicte, Maier, Esther M, Roubertie, Agathe, Santra, Saikat, Tylki-Szymanska, Anna, Day, Simon, Symonds, Tara, Hudgens, Stacie, Patterson, Marc C, Guldberg, Christina, Ingemann, Linda, Petersen, Nikolaj H. T, Kirkegaard, Thomas, Dali, Christine í

“…An amendment to this paper has been published and can be accessed via the original article…”
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Measurement properties of the Inclusion Body Myositis Functional Rating Scale by Salam, Sharfaraz, Symonds, Tara, Doll, Helen, Rousell, Sam, Randall, Jason, Lloyd-Price, Lucy, Hudgens, Stacie, Guldberg, Christina, Herbelin, Laura, Barohn, Richard J, Hanna, Michael G, Dimachkie, Mazen M, Machado, Pedro M

“…ObjectivesTo evaluate the validity, reliability, responsiveness and meaningful change threshold of the Inclusion Body Myositis (IBM) Functional Rating Scale…”
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Impacts and burden of Niemann-Pick disease type C: A patient and caregiver perspective by Mengel, Eugen, Patterson, Marc, Chladek, Michael, Guldberg, Christina, Dali, Christine i, Symonds, Tara, Lloyd-Price, Lucy, Mathieson, Toni, Crowe, Joslyn, Burbridge, Claire

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Validation of a short-form 5-domain Niemann-Pick type C clinical severity scale (5-domain NPCCSS) by Patterson, Marc, Lloyd-Price, Lucy, Guldberg, Christina, Hudgens, Stacie, Doll, Helen, Burbridge, Claire, Chladek, Michael, í Dali, Christine, Mengel, Eugen, Symonds, Tara

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Study to Assess Content Validity and Interrater and Intrarater Reliability of the Inclusion Body Myositis Functional Rating Scale by Symonds, Tara, Randall, Jason, Lloyd-Price, Lucy, Hudgens, Stacie, Dimachkie, Mazen M., Guldberg, Christina, Machado, Pedro M.

“…Sporadic inclusion body myositis (IBM) is a rare, muscle-wasting disease that negatively affects health-related quality of life. Although a measure that has…”
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Clinical disease progression and biomarkers in Niemann-Pick disease type C: a prospective cohort study by Mengel, Eugen, Bembi, Bruno, Del Toro, Mireia, Deodato, Federica, Gautschi, Matthias, Grunewald, Stephanie, Grønborg, Sabine, Héron, Bénédicte, Maier, Esther M, Roubertie, Agathe, Santra, Saikat, Tylki-Szymanska, Anna, Day, Simon, Symonds, Tara, Hudgens, Stacie, Patterson, Marc C, Guldberg, Christina, Ingemann, Linda, Petersen, Nikolaj H T, Kirkegaard, Thomas, Í Dali, Christine

“…Niemann-Pick disease type C (NPC) is a rare, progressive, neurodegenerative disease associated with neurovisceral manifestations resulting from lysosomal…”
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