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Enzyme replacement therapy

Skin globotriaosylceramide 3 deposits are specific to Fabry disease with classical mutations and associated with small fibre neuropathy

Rocco Liguori, Alex Incensi, [ ... ], Vincenzo Donadio

Efficacy and safety of intravenous laronidase for mucopolysaccharidosis type I: A systematic review and meta-analysis

Alícia Dorneles Dornelles, Osvaldo Artigalás, [ ... ], Ida Vanessa Doederlein Schwartz

Anti-TNF-Alpha Therapy Enhances the Effects of Enzyme Replacement Therapy in Rats with Mucopolysaccharidosis Type VI

Efrat Eliyahu, Theodore Wolfson, [ ... ], Calogera M. Simonaro

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

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

One Year of Enzyme Replacement Therapy Reduces Globotriaosylceramide Inclusions in Podocytes in Male Adult Patients with Fabry Disease

Behzad Najafian, Camilla Tøndel, [ ... ], Michael Mauer

Cardiomyopathy and Response to Enzyme Replacement Therapy in a Male Mouse Model for Fabry Disease

Aurelie Nguyen Dinh Cat, Brigitte Escoubet, [ ... ], Frederic Jaisser

A Distinct Urinary Biomarker Pattern Characteristic of Female Fabry Patients That Mirrors Response to Enzyme Replacement Therapy

Andreas D. Kistler, Justyna Siwy, [ ... ], Andreas L. Serra

Urinary Podocyte Loss Is Increased in Patients with Fabry Disease and Correlates with Clinical Severity of Fabry Nephropathy

Brent Fall, C. Ronald Scott, [ ... ], Behzad Najafian

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

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

Longitudinal study on ocular manifestations in a cohort of patients with Fabry disease

Langis Michaud

Time of Initiating Enzyme Replacement Therapy Affects Immune Abnormalities and Disease Severity in Patients with Gaucher Disease

Renuka Pudi Limgala, Chidima Ioanou, [ ... ], Ozlem Goker-Alpan

Specificity of a Polyclonal Fecal Elastase ELISA for CELA3

Frank Ulrich Weiss, Christoph Budde, Markus M. Lerch

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

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

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