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Enzyme replacement therapy

Quantification of intramuscular fat in patients with late-onset Pompe disease by conventional magnetic resonance imaging for the long-term follow-up of enzyme replacement therapy

André Lollert, Clemens Stihl, [ ... ], Gundula Staatz

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

Retrospective study of long-term outcomes of enzyme replacement therapy in Fabry disease: Analysis of prognostic factors

Maarten Arends, Marieke Biegstraaten, [ ... ], Carla E. M. Hollak

Long-term follow-up of pulmonary function in Fabry disease: A bi-center observational study

Daniel P. Franzen, Albina Nowak, [ ... ], Frederic Barbey

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

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

Enzyme replacement therapy for Anderson-Fabry disease: A complementary overview of a Cochrane publication through a linear regression and a pooled analysis of proportions from cohort studies

Regina El Dib, Huda Gomaa, [ ... ], Fellype Barreto

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

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

Skin Globotriaosylceramide 3 Load Is Increased in Men with Advanced Fabry Disease

Nurcan Üçeyler, Nils Schröter, [ ... ], Claudia Sommer

Uric Acid as a Marker of Mortality and Morbidity in Fabry Disease

Daniel Rob, Josef Marek, [ ... ], Aleš Linhart

Specificity of a Polyclonal Fecal Elastase ELISA for CELA3

Frank Ulrich Weiss, Christoph Budde, Markus M. Lerch

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

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

Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase

David G. Warnock, Daniel G. Bichet, [ ... ], Franklin K. Johnson

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