1-9 of 9
mucopolysaccharidosis i
Close
Save search
Follow your search
Access your saved searches in your account
Would you like to receive an alert when new items match your search?
Close Modal
Sort by
Journal Articles

Leveraging Evidence-Based Public Policy and Advocacy to Advance Newborn Screening in California

Max G. Bronstein, MPP, Richard J. Pan, MD, Mark Dant, Bertrand Lubin, MD
Pediatrics (2019) 143 (2): e20181886.
Published: February 2019
View article
PDF
Journal Articles

Delay in State Adoption of Newborn Screening Tests

Ang Xu, BA, Vignesh Ganapathy, JD, Stephanie R. Morain, MPH
Pediatrics (2018) 141 (1): e20170300.
Published: January 2018
View article
PDF
Journal Articles

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
View article
PDF
Journal Articles

Laronidase for Cardiopulmonary Disease in Hurler Syndrome 12 Years After Bone Marrow Transplantation

Vassili Valayannopoulos, MD, Jacques de Blic, MD, PhD, Nizar Mahlaoui, MD, Bertrand Stos, MD, Francis Jaubert, MD, PhD ...
Pediatrics (2010) 126 (5): e1242–e1247.
Published: November 2010
View article
PDF
Journal Articles

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
View article
PDF
Journal Articles

Mucopolysaccharidosis I: Management and Treatment Guidelines

Joseph Muenzer, MD, PhD, James E. Wraith, MB, ChB, Lorne A. Clarke, MD, and the International Consensus Panel on the Management and Treatment of Mucopolysaccharidosis I
Pediatrics (2009) 123 (1): 19–29.
Published: January 2009
View article
PDF
Journal Articles

Long-term Efficacy and Safety of Laronidase in the Treatment of Mucopolysaccharidosis I

Lorne A. Clarke, MD, J. Edmond Wraith, MB, ChB, Michael Beck, MD, Edwin H. Kolodny, MD, Gregory M. Pastores, MD ...
Pediatrics (2009) 123 (1): 229–240.
Published: January 2009
View article
PDF
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
View article
PDF
Journal Articles

A Characteristic Bone Marrow Finding in the Hurler Syndrome

Howard A. Pearson, Andrew E. Lorincz
Pediatrics (1964) 34 (2): 280–282.
Published: August 1964
View article
PDF