Metachromatic Leukodystrophy: An Assessment of Disease Burden von Eichler, Florian S., Cox, Timothy M., Crombez, Eric, Dali, Christine í, Kohlschütter, Alfried

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Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials von Cortina-Borja, Mario, Te Vruchte, Danielle, Mengel, Eugen, Amraoui, Yasmin, Imrie, Jackie, Jones, Simon A, I Dali, Christine, Fineran, Paul, Kirkegaard, Thomas, Runz, Heiko, Lachmann, Robin, Bremova-Ertl, Tatiana, Strupp, Michael, Platt, Frances M

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Impacts and Burden of Niemann pick Type-C: a patient and caregiver perspective von Mengel, Eugen, Patterson, Marc C, Chladek, Michael, Guldberg, Christina, Í Dali, Christine, Symonds, Tara, Lloyd-Price, Lucy, Mathieson, Toni, Crowe, Joslyn, Burbridge, Claire

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Efficacy and safety of arimoclomol in Niemann‐Pick disease type C: Results from a double‐blind, randomised, placebo‐controlled, multinational phase 2/3 trial of a novel treatment... von Mengel, Eugen, Patterson, Marc C., Da Riol, Rosalia M., Del Toro, Mireia, Deodato, Federica, Gautschi, Matthias, Grunewald, Stephanie, Grønborg, Sabine, Harmatz, Paul, Héron, Bénédicte, Maier, Esther M., Roubertie, Agathe, Santra, Saikat, Tylki‐Szymanska, Anna, Day, Simon, Andreasen, Anne Katrine, Geist, Marie Aavang, Havnsøe Torp Petersen, Nikolaj, Ingemann, Linda, Hansen, Thomas, Blaettler, Thomas, Kirkegaard, Thomas, Dali, Christine

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Cerebral gray and white matter changes and clinical course in metachromatic leukodystrophy von GROESCHEL, Samuel, DALI, Christine I, CLAS, Philipp, BÖHRINGER, Judith, DUNO, Morten, KRARUP, Christian, KEHRER, Christiane, WILKE, Marko, KRÄGELOH-MANN, Ingeborg

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Metachromatic leukodystrophy: natural course of cerebral MRI changes in relation to clinical course von Groeschel, Samuel, Kehrer, Christiane, Engel, Corinna, í Dali, Christine, Bley, Annette, Steinfeld, Robert, Grodd, Wolfgang, Krägeloh-Mann, Ingeborg

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Clinical disease progression and biomarkers in Niemann-Pick disease type C: a prospective cohort study von 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

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Intravenous arylsulfatase A in metachromatic leukodystrophy: a phase 1/2 study von í Dali, Christine, Groeschel, Samuel, Moldovan, Mihai, Farah, Mohamed H., Krägeloh‐Mann, Ingeborg, Wasilewski, Margaret, Li, Jing, Barton, Norman, Krarup, Christian

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Effects of sulfatide on peripheral nerves in metachromatic leukodystrophy von Farah, Mohamed H., Dali, Christine í, Groeschel, Samuel, Moldovan, Mihai, Whiteman, David A. H., Malanga, C. J., Krägeloh‐Mann, Ingeborg, Li, Jing, Barton, Norman, Krarup, Christian

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Correction to: Clinical disease progression and biomarkers in Niemann–Pick disease type C: a prospective cohort study von 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

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Alternating hemiplegia of childhood in Denmark: Clinical manifestations and ATP1A3 mutation status von Hoei-Hansen, Christina E, Dali, Christine í, Lyngbye, Troels J.B, Duno, Morten, Uldall, Peter

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Erratum to: Review and evaluation of the methodological quality of the existing guidelines and recommendations for inherited neurometabolic disorders von Cassis, Linda, Cortès-Saladelafont, Elisenda, Molero-Luis, Marta, Yubero, Delia, González, Maria Julieta, Ormazábal, Aida, Fons, Carme, Jou, Cristina, Sierra, Cristina, Ponce, Esperanza Castejon, Ramos, Federico, Armstrong, Judith, O'Callaghan, M Mar, Casado, Mercedes, Montero, Raquel, Meavilla-Olivas, Silvia, Artuch, Rafael, Barić, Ivo, Bartoloni, Franco, Bellettato, Cinzia Maria, Bonifazi, Fedele, Ceci, Adriana, Cvitanović-Šojat, Ljerka, Dali, Christine I, D'Avanzo, Francesca, Fumic, Ksenija, Giannuzzi, Viviana, Lampe, Christina, Scarpa, Maurizio, Garcia-Cazorla, Ángels

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Safety of intrathecal delivery of recombinant human arylsulfatase A in children with metachromatic leukodystrophy: Results from a phase 1/2 clinical trial von í Dali, Christine, Sevin, Caroline, Krägeloh-Mann, Ingeborg, Giugliani, Roberto, Sakai, Norio, Wu, James, Wasilewski, Margaret

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Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-bl... von Borgwardt, Line, Guffon, Nathalie, Amraoui, Yasmina, Dali, Christine I., De Meirleir, Linda, Gil-Campos, Mercedes, Heron, Bénédicte, Geraci, Silvia, Ardigò, Diego, Cattaneo, Federica, Fogh, Jens, Van den Hout, J. M. Hannerieke, Beck, Michael, Jones, Simon A., Tylki-Szymanska, Anna, Haugsted, Ulla, Lund, Allan M.

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Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis von Lund, Allan M., Borgwardt, Line, Cattaneo, Federica, Ardigò, Diego, Geraci, Silvia, Gil-Campos, Mercedes, De Meirleir, Linda, Laroche, Cécile, Dolhem, Philippe, Cole, Duncan, Tylki-Szymanska, Anna, Lopez-Rodriguez, Monica, Guillén-Navarro, Encarna, Dali, Christine I., Héron, Bénédicte, Fogh, Jens, Muschol, Nicole, Phillips, Dawn, Van den Hout, J. M. Hannerieke, Jones, Simon A., Amraoui, Yasmina, Harmatz, Paul, Guffon, Nathalie

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Alpha-mannosidosis: correlation between phenotype, genotype and mutant MAN2B1 subcellular localisation von Borgwardt, Line, Stensland, Hilde Monica Frostad Riise, Olsen, Klaus Juul, Wibrand, Flemming, Klenow, Helle Bagterp, Beck, Michael, Amraoui, Yasmina, Arash, Laila, Fogh, Jens, Nilssen, Øivind, Dali, Christine I, Lund, Allan Meldgaard

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Arimoclomol reduces levels of biomarkers of lipid burden in patients with Niemann-Pick disease type C von Andersen, Lene, Petersen, Nikolaj H., Ingemann, Linda, Fog, Cathrine K., Dali, Christine í, Kirkegaard, Thomas

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Cowden Syndrome and Concomitant Pulmonary Neuroendocrine Tumor: A Presentation of Two Cases von Knigge, Ulrich Peter, Gerdes, Anne-Marie, Rasmussen, Åse Krogh, Petersen, Rene Horsleben, Dali, Christine I., Ringholm, Lene, Langer, Seppo W., Federspiel, Birgitte

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Effects of sulfatide accumulation on peripheral nerves in metachromatic leukodystrophy (P3-5.001) von Krarup, Christian, Dali, Christine í, Groeschel, Samuel, Moldovan, Mihai, Whiteman, David A.H., Krägeloh-Mann, Ingeborg, Li, Jing, Barton, Norman, Farah, Mohamed H.

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The ethical framework for performing research with rare inherited neurometabolic disease patients von Giannuzzi, Viviana, Devlieger, Hugo, Margari, Lucia, Odlind, Viveca Lena, Ragab, Lamis, Bellettato, Cinzia Maria, D’Avanzo, Francesca, Lampe, Christina, Cassis, Linda, Cortès-Saladelafont, Elisenda, Cazorla, Ángels Garcia, Barić, Ivo, Cvitanović-Šojat, Ljerka, Fumić, Ksenija, Dali, Christine I, Bartoloni, Franco, Bonifazi, Fedele, Scarpa, Maurizio, Ceci, Adriana

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