Mucopolysaccharidosis VI: pathophysiology, diagnosis and treatment von Harmatz, Paul, Shediac, Renee

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Mucopolysaccharidosis VI von Valayannopoulos, Vassili, Nicely, Helen, Harmatz, Paul, Turbeville, Sean

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Enzyme Replacement Therapies and Immunogenicity in Lysosomal Storage Diseases: Is There a Pattern? von Harmatz, Paul, MD

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Current and new therapies for mucopolysaccharidoses von Penon-Portmann, Monica, Blair, David R., Harmatz, Paul

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Management Guidelines for Mucopolysaccharidosis VI von Giugliani, Roberto, Harmatz, Paul, Wraith, James E

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Neurodevelopmental status and adaptive behavior of pediatric patients with mucopolysaccharidosis II: a longitudinal observational study von Muenzer, Joseph, Burton, Barbara K, Amartino, Hernan M, Harmatz, Paul R, Gutiérrez-Solana, Luis González, Ruiz-Garcia, Matilde, Wu, Yuna, Merberg, David, Alexanderian, David, Jones, Simon A

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Mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome): defining and measuring functional impacts in pediatric patients von Leiro, Beth, Phillips, Dawn, Duiker, Melanie, Harmatz, Paul, Charles, Sharon

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Evaluation of the long-term treatment effects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling: data from the Hunter Outcome... von Muenzer, Joseph, Botha, Jaco, Harmatz, Paul, Giugliani, Roberto, Kampmann, Christoph, Burton, Barbara K

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Surgical management of neurological manifestations of mucopolysaccharidosis disorders von Alden, Tord D., Amartino, Hernán, Dalla Corte, Amauri, Lampe, Christina, Harmatz, Paul R., Vedolin, Leonardo

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Recognition and Diagnosis of Mucopolysaccharidosis II (Hunter Syndrome) von Martin, Rick, Beck, Michael, Eng, Christine, Giugliani, Roberto, Harmatz, Paul, Munoz, Veronica, Muenzer, Joseph

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Cardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and management von Braunlin, Elizabeth A., Harmatz, Paul R., Scarpa, Maurizio, Furlanetto, Beatriz, Kampmann, Christoph, Loehr, James P., Ponder, Katherine P., Roberts, William C., Rosenfeld, Howard M., Giugliani, Roberto

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Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance von Akyol, Mehmet Umut, Alden, Tord D, Amartino, Hernan, Ashworth, Jane, Belani, Kumar, Berger, Kenneth I, Borgo, Andrea, Braunlin, Elizabeth, Eto, Yoshikatsu, Gold, Jeffrey I, Jester, Andrea, Jones, Simon A, Karsli, Cengiz, Mackenzie, William, Marinho, Diane Ruschel, McFadyen, Andrew, McGill, Jim, Mitchell, John J, Muenzer, Joseph, Okuyama, Torayuki, Orchard, Paul J, Stevens, Bob, Thomas, Sophie, Walker, Robert, Wynn, Robert, Giugliani, Roberto, Harmatz, Paul, Hendriksz, Christian, Scarpa, Maurizio

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Spinal involvement in mucopolysaccharidosis IVA (Morquio-Brailsford or Morquio A syndrome): presentation, diagnosis and management von Solanki, Guirish A., Martin, Kenneth W., Theroux, Mary C., Lampe, Christina, White, Klane K., Shediac, Renée, Lampe, Christian G., Beck, Michael, Mackenzie, William G., Hendriksz, Christian J., Harmatz, Paul R.

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International guidelines for the management and treatment of Morquio A syndrome von Hendriksz, Christian J., Berger, Kenneth I., Giugliani, Roberto, Harmatz, Paul, Kampmann, Christoph, Mackenzie, William G., Raiman, Julian, Villarreal, Martha Solano, Savarirayan, Ravi

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Non-invasive assessment of tissue iron overload von Fischer, Roland, Harmatz, Paul R

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Clinical characteristics and somatic burden of patients with mucopolysaccharidosis II with or without neurological involvement: An analysis from the Hunter Outcome Survey von Lau, Heather, Harmatz, Paul, Botha, Jaco, Audi, Jennifer, Link, Bianca

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A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B von Muschol, Nicole, Koehn, Anja, von Cossel, Katharina, Okur, Ilyas, Ezgu, Fatih, Harmatz, Paul, de Castro Lopez, Maria J, Couce, Maria Luz, Lin, Shuan-Pei, Batzios, Spyros, Cleary, Maureen, Solano, Martha, Nestrasil, Igor, Kaufman, Brian, Shaywitz, Adam J, Maricich, Stephen M, Kuca, Bernice, Kovalchin, Joseph, Zanelli, Eric

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Anaesthesia and airway management in mucopolysaccharidosis von Walker, Robert, Belani, Kumar G., Braunlin, Elizabeth A., Bruce, Iain A., Hack, Henrik, Harmatz, Paul R., Jones, Simon, Rowe, Richard, Solanki, Guirish A., Valdemarsson, Barbara

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Enzyme replacement therapy initiated in adulthood: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program von Lampe, Christina, Harmatz, Paul R., Parini, Rossella, Sharma, Reena, Teles, Elisa Leão, Johnson, Julie, Sivam, Debbie, Sisic, Zlatko

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Assessing the impact of the five senses on quality of life in mucopolysaccharidoses von Giugliani, Roberto, Harmatz, Paul, Lin, Shuan-Pei, Scarpa, Maurizio

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