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Journal Articles

Hypertrophic Cardiomyopathy in Infantile-Onset Pompe Disease Treated with Enzyme Replacement Therapy

Sabena F. Hussain, MD, Gabrielle Geddes, MD, Marcus Schamberger, MD, John Parent, MD, MSCR
Pediatrics (2021) 147 (3_MeetingAbstract): 350–351.
Published: March 2021
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Journal Articles

A 24-Year Enzyme Replacement Therapy in an Adenosine-deaminase-Deficient Patient

Hana M. Tartibi, MD, Michael S. Hershfield, MD, Sami L. Bahna, MD
Pediatrics (2016) 137 (1): e20152169.
Published: January 2016
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Fatal Coronary Artery Disease in an Infant With Severe Mucopolysaccharidosis Type I

Leonie van den Broek, MD, Ad P. C. M. Backx, MD, Hans Coolen, Frits A. Wijburg, MD, PhD, Ron Wevers, MD, PhD ...
Pediatrics (2011) 127 (5): e1343–e1346.
Published: May 2011
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Enzyme-Replacement Therapy in a 5-Month-Old Boy With Attenuated Presymptomatic MPS I: 5-Year Follow-up

Orazio Gabrielli, MD, Lorne A. Clarke, MD, Stefano Bruni, MD, Giovanni V. Coppa, MD
Pediatrics (2010) 125 (1): e183–e187.
Published: January 2010
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Eight-Year Clinical Outcomes of Long-Term Enzyme Replacement Therapy for 884 Children With Gaucher Disease Type 1

Hans Andersson, MD, Paige Kaplan, MBBCh, Katherine Kacena, PhD, John Yee, MD, MPH
Pediatrics (2008) 122 (6): 1182–1190.
Published: December 2008
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A Prospective, Cross-sectional Survey Study of the Natural History of Niemann-Pick Disease Type B

Margaret M. McGovern, MD, PhD, Melissa P. Wasserstein, MD, Roberto Giugliani, MD, PhD, Bruno Bembi, MD, Marie T. Vanier, MD, PhD ...
Pediatrics (2008) 122 (2): e341–e349.
Published: August 2008
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Journal Articles

Successful Management of Difficult Infusion-Associated Reactions in a Young Patient With Mucopolysaccharidosis Type VI Receiving Recombinant Human Arylsulfatase B (Galsulfase [Naglazyme])

Katherine H. Kim, MS, Celeste Decker, MD, Barbara K. Burton, MD
Pediatrics (2008) 121 (3): e714–e717.
Published: March 2008
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Journal Articles

Enzyme Replacement Therapy in Patients Who Have Mucopolysaccharidosis I and Are Younger Than 5 Years: Results of a Multinational Study of Recombinant Human α-l-Iduronidase (Laronidase)

J. Edmond Wraith, MBChB, Michael Beck, MD, Roderick Lane, PhD, Ans van der Ploeg, MD, PhD, Elsa Shapiro, PhD, ABPP ...
Pediatrics (2007) 120 (1): e37–e46.
Published: July 2007
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Journal Articles

Enzyme-Replacement Therapy With Agalsidase Alfa in Children With Fabry Disease

Markus Ries, MD, MHSc, Joe T.R. Clarke, MD, PhD, Catharina Whybra, MD, Margaret Timmons, MD, Chevalia Robinson, RN, BSN ...
Pediatrics (2006) 118 (3): 924–932.
Published: September 2006
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Journal Articles

Direct Comparison of Measures of Endurance, Mobility, and Joint Function During Enzyme-Replacement Therapy of Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome): Results After 48 Weeks in a Phase 2 Open-Label Clinical Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase

Paul Harmatz, MD, David Ketteridge, MBBS, Roberto Giugliani, MD, PhD, Natalie Guffon, MD, Elisa Leão Teles, MD ...
Pediatrics (2005) 115 (6): e681–e689.
Published: June 2005
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Journal Articles

Nephrotic Syndrome Complicating α-Glucosidase Replacement Therapy for Pompe Disease

Tracy E. Hunley, MD, Deyanira Corzo, MD, Martha Dudek, MS, Priya Kishnani, MD, Andrea Amalfitano, DO, PhD ...
Pediatrics (2004) 114 (4): e532–e535.
Published: October 2004
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Journal Articles

The Natural Course of Infantile Pompe’s Disease: 20 Original Cases Compared With 133 Cases From the Literature

Hannerieke M. P. van den Hout, Wim Hop, Otto P. van Diggelen, Jan A. M. Smeitink, G. Peter A. Smit ...
Pediatrics (2003) 112 (2): 332–340.
Published: August 2003
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Gaucher’s Disease and Fatal Hepatic Fibrosis Despite Prolonged Enzyme Replacement Therapy

Yves Perel, MD, Paulette Bioulac-Sage, MD, Jean-François Chateil, MD, Herve Trillaud, MD, Jacques Carles, MD ...
Pediatrics (2002) 109 (6): 1170–1173.
Published: June 2002
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Enzyme Replacement Therapy for Gaucher Disease: Skeletal Responses to Macrophage-targeted Glucocerebrosidase

Daniel I. Rosenthal, Samuel H. Doppelt, Henry J. Mankin, James M. Dambrosia, Ramnik J. Xavier ...
Pediatrics (1995) 96 (4): 629–637.
Published: October 1995
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