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Enzyme replacement therapy

Rapid Immunochromatographic Detection of Serum Anti-α-Galactosidase A Antibodies in Fabry Patients after Enzyme Replacement Therapy

Sachie Nakano, Takahiro Tsukimura, [ ... ], Futoshi Shibasaki

Pharmacokinetics of Novel Plant Cell-Expressed Taliglucerase Alfa in Adult and Pediatric Patients with Gaucher Disease

Richat Abbas, Glen Park, [ ... ], Sari Alon

Effects of Enzyme Replacement Therapy Started Late in a Murine Model of Mucopolysaccharidosis Type I

Gabriela Pasqualim, Guilherme Baldo, [ ... ], Ursula Matte

Mosaicism of Podocyte Involvement Is Related to Podocyte Injury in Females with Fabry Disease

Michael Mauer, Emily Glynn, [ ... ], Behzad Najafian

Increased Arterial Diameters in the Posterior Cerebral Circulation in Men with Fabry Disease

Nurcan Üçeyler, György A. Homola, [ ... ], Claudia Sommer

The Effect of Recombinant Human Iduronate-2-Sulfatase (Idursulfase) on Growth in Young Patients with Mucopolysaccharidosis Type II

Zbigniew Żuber, Agnieszka Różdżyńska-Świątkowska, Agnieszka Jurecka, Anna Tylki-Szymańska

Glucosylsphingosine Is a Highly Sensitive and Specific Biomarker for Primary Diagnostic and Follow-Up Monitoring in Gaucher Disease in a Non-Jewish, Caucasian Cohort of Gaucher Disease Patients

Arndt Rolfs, Anne-Katrin Giese, [ ... ], Hermann Mascher

Myostatin and Insulin-Like Growth Factor I: Potential Therapeutic Biomarkers for Pompe Disease

Yin-Hsiu Chien, Der-Sheng Han, [ ... ], Wei-Shiung Yang

A Rapid and Sensitive Method for Measuring N-Acetylglucosaminidase Activity in Cultured Cells

Victor Mauri, Parisa Lotfi, Laura Segatori, Marco Sardiello

Algorithm for the Early Diagnosis and Treatment of Patients with Cross Reactive Immunologic Material-Negative Classic Infantile Pompe Disease: A Step towards Improving the Efficacy of ERT

Suhrad G. Banugaria, Sean N. Prater, [ ... ], Priya S. Kishnani

Illness Perception and Clinical Treatment Experiences in Patients with M. Maroteaux-Lamy (Mucopolysaccharidosis Type VI) and a Turkish Migration Background in Germany

Hansjörg Dilger, Linn Leissner, [ ... ], Ursula Plöckinger

Liver Fibrosis in Type I Gaucher Disease: Magnetic Resonance Imaging, Transient Elastography and Parameters of Iron Storage

Anneloes E. Bohte, Laura van Dussen, [ ... ], Carla E. M. Hollak

Long-Term Effect of Antibodies against Infused Alpha-Galactosidase A in Fabry Disease on Plasma and Urinary (lyso)Gb3 Reduction and Treatment Outcome

Saskia M. Rombach, Johannes M. F. G. Aerts, [ ... ], Gabor E. Linthorst

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