Reversed papilledema in an MPS VI patient with galsulfase (Naglazyme(®)) therapy

Autor Koseoglu, Selim T., Harmatz, Paul, Turbeville, Sean, Nicely, Helen
Vydáno 2008

Mucopolysaccharidosis VI

Autor Valayannopoulos, Vassili, Nicely, Helen, Harmatz, Paul, Turbeville, Sean
Vydáno 2010

Assessing the impact of the five senses on quality of life in mucopolysaccharidoses

Autor Giugliani, Roberto, Harmatz, Paul, Lin, Shuan-Pei, Scarpa, Maurizio
Vydáno v Orphanet J Rare Dis (2020)

Italian Society of Hematology guidelines for thalassemia and non-invasive iron measurements

Autor Nielsen, Peter, Engelhardt, Rainer, Grosse, Regine, Janka, Gritta, Harmatz, Paul, Fischer, Roland
Vydáno 2009

Bone density assessment in patients with mucopolysaccharidosis: A preliminary report from patients with MPS II and VI

Autor Fung, Ellen B., Johnson, Jo Ann, Madden, Jacqueline, Kim, Tiffany, Harmatz, Paul
Vydáno 2010

Risks of long-term port use in enzyme replacement therapy for lysosomal storage disorders

Autor Hendriksz, Christian J., Harmatz, Paul, Giugliani, Roberto, Roberts, Jane, Arul, G. Suren
Vydáno v Mol Genet Metab Rep (2018)

Growth Charts for Individuals with Mucopolysaccharidosis VI (Maroteaux–Lamy Syndrome)

Autor Quartel, Adrian, Hendriksz, Christian J., Parini, Rossella, Graham, Sue, Lin, Ping, Harmatz, Paul
Vydáno v JIMD Rep (2014)

Intrafamilial variability in the clinical manifestations of mucopolysaccharidosis type II: Data from the Hunter Outcome Survey (HOS)

Autor Ficicioglu, Can, Giugliani, Roberto, Harmatz, Paul, Mendelsohn, Nancy J., Jego, Virginie, Parini, Rossella
Vydáno v Am J Med Genet A (2017)

Pulmonary Hypertension In Well-Transfused Thalassemia Major Patients

Autor Meloni, Antonella, Detterich, Jon, Pepe, Alessia, Harmatz, Paul, Coates, Tom D, Wood, John C.
Vydáno v Blood Cells Mol Dis (2014)

Electrocardiographic consequences of cardiac iron overload in thalassemia major

Autor Detterich, Jon, Noetzli, Leila, Dorey, Fred, Bar-Cohen, Yaniv, Harmatz, Paul, Coates, Thomas, Wood, John
Vydáno 2011

The Economic Burden of Home Care for Children with HIV and Other Chronic Illnesses

Autor Wilson, Leslie S., Moskowitz, Judith Tedlie, Acree, Michael, Heyman, Melvin B., Harmatz, Paul, Ferrando, Stephen J., Folkman, Susan
Vydáno 2005

Combined Chelation Therapy with Deferasirox and Deferoxamine in Thalassemia

Autor Lal, Ashutosh, Porter, John, Sweeters, Nancy, Ng, Vivian, Evans, Patricia, Neumayr, Lynne, Kurio, Gregory, Harmatz, Paul, Vichinsky, Elliott
Vydáno 2012

Development of a Scoring System to Evaluate the Severity of Craniocervical Spinal Cord Compression in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)

Autor Möllmann, Christian, Lampe, Christian G., Müller-Forell, Wibke, Scarpa, Maurizio, Harmatz, Paul, Schwarz, Manfred, Beck, Michael, Lampe, Christina
Vydáno 2013

Galsulfase (Naglazyme®) therapy in infants with mucopolysaccharidosis VI

Autor Harmatz, Paul R., Garcia, Paula, Guffon, Nathalie, Randolph, Linda M., Shediac, Renée, Braunlin, Elizabeth, Lachman, Ralph S., Decker, Celeste
Vydáno 2013

Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y

Autor Jones, Simon A., Bialer, Martin, Parini, Rossella, Martin, Ken, Wang, Hui, Yang, Ke, Shaywitz, Adam J., Harmatz, Paul
Vydáno v Pediatr Res (2015)

Relationship between labile plasma iron, liver iron concentration and cardiac response in a deferasirox monotherapy trial

Autor Wood, John C., Glynos, Tara, Thompson, Alexis, Giardina, Patricia, Harmatz, Paul, Kang, Barinder P., Paley, Carole, Coates, Thomas D.
Vydáno 2011

Orthopedic management of the extremities in patients with Morquio A syndrome

Autor White, Klane K., Jester, Andrea, Bache, C. Edward, Harmatz, Paul R., Shediac, Renée, Thacker, Mihir M., Mackenzie, William G.
Vydáno 2014

Individual heat map assessments demonstrate vestronidase alfa treatment response in a highly heterogeneous mucopolysaccharidosis VII study population

Autor Haller, Christine, Song, Wenjie, Cimms, Tricia, Chen, Chao‐Yin, Whitley, Chester B., Wang, Raymond Y., Bauer, Mislen, Harmatz, Paul
Vydáno v JIMD Rep (2019)

Pulmonary function in thalassaemia major and its correlation with body iron stores

Autor Sohn, Eugene Y, Noetzli, Leila J, Gera, Aakanksha, Kato, Roberta, Coates, Thomas D., Harmatz, Paul, Keens, Thomas G, Wood, John C
Vydáno 2011

The Immune Response to Intrathecal Enzyme Replacement Therapy in Mucopolysaccharidosis I Patients

Autor Vera, Moin, Le, Steven, Kan, Shih-hsin, Garban, Hermes, Naylor, David, Mlikotic, Anton, Kaitila, Ilkka, Harmatz, Paul, Chen, Agnes, Dickson, Patricia
Vydáno 2013