Marsha Treadwell
UCSF Benioff Children’s Hospital Oakland, Oakland, CA, USAUCSFUSAOakland, CA, USAUCSF Benioff Children’s Hospital Oakland, Oakland, CA, USA
Authors’ Contributions
MT was consultant to the study, designed the plan for the pain assessment strategies used in the present study, scored and interpreted the pain assessments, and reviewed data analyses. PRH, BKB, JJM, NM, SAJ, GMP, HAL, RS, and VRS were all primary investigators of this study, contributed to the planning of the study, were involved in the clinical examinations and collection of data. JIG provided insight in the interpretation of the data. CH and AJS assisted with conduction of the study, data analyses, and development of the manuscript. All authors contributed to the writing and critical review of the manuscript and approved the final manuscript.
Declaration of Conflicting Interests
The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Marsha Treadwell received consulting fees and travel and meeting reimbursement from BioMarin.
Paul R. Harmatz received consulting fees from BioMarin, Shire, Genzyme, Alexion, Chiesi, Inventiva, PTC, and ReGenX Bio; he received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
Barbara K. Burton received consulting fees from BioMarin, Shire, Genzyme, Horizon Pharma, Alexion, and ReGenX Bio; she received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, Cytonet, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
John J. Mitchell received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, and Shire.
Nicole Muschol received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Amicus.
Simon A. Jones worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received a honorarium.
Gregory M. Pastores received consulting fees from BioMarin, Genzyme, Pfizer, and Shire.
Heather A. Lau received consulting fees from Genzyme, Pfizer, and BioMarin; she received research funding from Genzyme, NTSAD, BioMarin, Ultragenyx, Pfizer, Shire, Amicus, and GSK and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Pfizer.
Rebecca Sparkes received consulting fees, research funding, and travel and meeting reimbursement from BioMarin.
V. Reid Sutton worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received an honorarium.
Christine Haller is a former employee of BioMarin Pharmaceutical Inc. and a current employee of Ultragenyx.
Bianca Meesen is an employee of Ismar Healthcare NV.
Adam J. Shaywitz is an employee of BioMarin Pharmaceutical Inc. and holder of stock options.
Jeffrey I. Gold received speaker fees and travel reimbursement from BioMarin.
Paul R. Harmatz
UCSF Benioff Children’s Hospital Oakland, Oakland, CA, USAUCSFUSAOakland, CA, USAUCSF Benioff Children’s Hospital Oakland, Oakland, CA, USA
Authors’ Contributions
MT was consultant to the study, designed the plan for the pain assessment strategies used in the present study, scored and interpreted the pain assessments, and reviewed data analyses. PRH, BKB, JJM, NM, SAJ, GMP, HAL, RS, and VRS were all primary investigators of this study, contributed to the planning of the study, were involved in the clinical examinations and collection of data. JIG provided insight in the interpretation of the data. CH and AJS assisted with conduction of the study, data analyses, and development of the manuscript. All authors contributed to the writing and critical review of the manuscript and approved the final manuscript.
Declaration of Conflicting Interests
The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Marsha Treadwell received consulting fees and travel and meeting reimbursement from BioMarin.
Paul R. Harmatz received consulting fees from BioMarin, Shire, Genzyme, Alexion, Chiesi, Inventiva, PTC, and ReGenX Bio; he received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
Barbara K. Burton received consulting fees from BioMarin, Shire, Genzyme, Horizon Pharma, Alexion, and ReGenX Bio; she received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, Cytonet, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
John J. Mitchell received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, and Shire.
Nicole Muschol received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Amicus.
Simon A. Jones worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received a honorarium.
Gregory M. Pastores received consulting fees from BioMarin, Genzyme, Pfizer, and Shire.
Heather A. Lau received consulting fees from Genzyme, Pfizer, and BioMarin; she received research funding from Genzyme, NTSAD, BioMarin, Ultragenyx, Pfizer, Shire, Amicus, and GSK and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Pfizer.
Rebecca Sparkes received consulting fees, research funding, and travel and meeting reimbursement from BioMarin.
V. Reid Sutton worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received an honorarium.
Christine Haller is a former employee of BioMarin Pharmaceutical Inc. and a current employee of Ultragenyx.
Bianca Meesen is an employee of Ismar Healthcare NV.
Adam J. Shaywitz is an employee of BioMarin Pharmaceutical Inc. and holder of stock options.
Jeffrey I. Gold received speaker fees and travel reimbursement from BioMarin.
Barbara K. Burton
Ann and Robert H. Lurie Children’s Hospital, Northwestern University Feinberg School of Medicine, Chicago, IL, USANorthwestern University Feinberg School of MedicineUSAChicago, IL, USAAnn and Robert H. Lurie Children’s Hospital, Northwestern University Feinberg School of Medicine, Chicago, IL, USA
Authors’ Contributions
MT was consultant to the study, designed the plan for the pain assessment strategies used in the present study, scored and interpreted the pain assessments, and reviewed data analyses. PRH, BKB, JJM, NM, SAJ, GMP, HAL, RS, and VRS were all primary investigators of this study, contributed to the planning of the study, were involved in the clinical examinations and collection of data. JIG provided insight in the interpretation of the data. CH and AJS assisted with conduction of the study, data analyses, and development of the manuscript. All authors contributed to the writing and critical review of the manuscript and approved the final manuscript.
Declaration of Conflicting Interests
The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Marsha Treadwell received consulting fees and travel and meeting reimbursement from BioMarin.
Paul R. Harmatz received consulting fees from BioMarin, Shire, Genzyme, Alexion, Chiesi, Inventiva, PTC, and ReGenX Bio; he received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
Barbara K. Burton received consulting fees from BioMarin, Shire, Genzyme, Horizon Pharma, Alexion, and ReGenX Bio; she received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, Cytonet, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
John J. Mitchell received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, and Shire.
Nicole Muschol received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Amicus.
Simon A. Jones worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received a honorarium.
Gregory M. Pastores received consulting fees from BioMarin, Genzyme, Pfizer, and Shire.
Heather A. Lau received consulting fees from Genzyme, Pfizer, and BioMarin; she received research funding from Genzyme, NTSAD, BioMarin, Ultragenyx, Pfizer, Shire, Amicus, and GSK and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Pfizer.
Rebecca Sparkes received consulting fees, research funding, and travel and meeting reimbursement from BioMarin.
V. Reid Sutton worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received an honorarium.
Christine Haller is a former employee of BioMarin Pharmaceutical Inc. and a current employee of Ultragenyx.
Bianca Meesen is an employee of Ismar Healthcare NV.
Adam J. Shaywitz is an employee of BioMarin Pharmaceutical Inc. and holder of stock options.
Jeffrey I. Gold received speaker fees and travel reimbursement from BioMarin.
John J. Mitchell
Montreal Children’s Hospital, Quebec, CanadaMontreal Children’s HospitalCanadaQuebec, CanadaMontreal Children’s Hospital, Quebec, Canada
Authors’ Contributions
MT was consultant to the study, designed the plan for the pain assessment strategies used in the present study, scored and interpreted the pain assessments, and reviewed data analyses. PRH, BKB, JJM, NM, SAJ, GMP, HAL, RS, and VRS were all primary investigators of this study, contributed to the planning of the study, were involved in the clinical examinations and collection of data. JIG provided insight in the interpretation of the data. CH and AJS assisted with conduction of the study, data analyses, and development of the manuscript. All authors contributed to the writing and critical review of the manuscript and approved the final manuscript.
Declaration of Conflicting Interests
The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Marsha Treadwell received consulting fees and travel and meeting reimbursement from BioMarin.
Paul R. Harmatz received consulting fees from BioMarin, Shire, Genzyme, Alexion, Chiesi, Inventiva, PTC, and ReGenX Bio; he received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
Barbara K. Burton received consulting fees from BioMarin, Shire, Genzyme, Horizon Pharma, Alexion, and ReGenX Bio; she received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, Cytonet, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
John J. Mitchell received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, and Shire.
Nicole Muschol received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Amicus.
Simon A. Jones worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received a honorarium.
Gregory M. Pastores received consulting fees from BioMarin, Genzyme, Pfizer, and Shire.
Heather A. Lau received consulting fees from Genzyme, Pfizer, and BioMarin; she received research funding from Genzyme, NTSAD, BioMarin, Ultragenyx, Pfizer, Shire, Amicus, and GSK and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Pfizer.
Rebecca Sparkes received consulting fees, research funding, and travel and meeting reimbursement from BioMarin.
V. Reid Sutton worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received an honorarium.
Christine Haller is a former employee of BioMarin Pharmaceutical Inc. and a current employee of Ultragenyx.
Bianca Meesen is an employee of Ismar Healthcare NV.
Adam J. Shaywitz is an employee of BioMarin Pharmaceutical Inc. and holder of stock options.
Jeffrey I. Gold received speaker fees and travel reimbursement from BioMarin.
Nicole Muschol
University Medical Center Hamburg-Eppendorf, Hamburg, GermanyUniversity Medical Center Hamburg-EppendorfGermanyHamburg, GermanyUniversity Medical Center Hamburg-Eppendorf, Hamburg, Germany
Authors’ Contributions
MT was consultant to the study, designed the plan for the pain assessment strategies used in the present study, scored and interpreted the pain assessments, and reviewed data analyses. PRH, BKB, JJM, NM, SAJ, GMP, HAL, RS, and VRS were all primary investigators of this study, contributed to the planning of the study, were involved in the clinical examinations and collection of data. JIG provided insight in the interpretation of the data. CH and AJS assisted with conduction of the study, data analyses, and development of the manuscript. All authors contributed to the writing and critical review of the manuscript and approved the final manuscript.
Declaration of Conflicting Interests
The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Marsha Treadwell received consulting fees and travel and meeting reimbursement from BioMarin.
Paul R. Harmatz received consulting fees from BioMarin, Shire, Genzyme, Alexion, Chiesi, Inventiva, PTC, and ReGenX Bio; he received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
Barbara K. Burton received consulting fees from BioMarin, Shire, Genzyme, Horizon Pharma, Alexion, and ReGenX Bio; she received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, Cytonet, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
John J. Mitchell received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, and Shire.
Nicole Muschol received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Amicus.
Simon A. Jones worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received a honorarium.
Gregory M. Pastores received consulting fees from BioMarin, Genzyme, Pfizer, and Shire.
Heather A. Lau received consulting fees from Genzyme, Pfizer, and BioMarin; she received research funding from Genzyme, NTSAD, BioMarin, Ultragenyx, Pfizer, Shire, Amicus, and GSK and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Pfizer.
Rebecca Sparkes received consulting fees, research funding, and travel and meeting reimbursement from BioMarin.
V. Reid Sutton worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received an honorarium.
Christine Haller is a former employee of BioMarin Pharmaceutical Inc. and a current employee of Ultragenyx.
Bianca Meesen is an employee of Ismar Healthcare NV.
Adam J. Shaywitz is an employee of BioMarin Pharmaceutical Inc. and holder of stock options.
Jeffrey I. Gold received speaker fees and travel reimbursement from BioMarin.
Simon A. Jones
Willink Unit, St. Mary’s Hospital, CMFT, MAHSC, University of Manchester, Manchester, United KingdomUniversity of ManchesterUnited KingdomManchester, United KingdomWillink Unit, St. Mary’s Hospital, CMFT, MAHSC, University of Manchester, Manchester, United Kingdom
Authors’ Contributions
MT was consultant to the study, designed the plan for the pain assessment strategies used in the present study, scored and interpreted the pain assessments, and reviewed data analyses. PRH, BKB, JJM, NM, SAJ, GMP, HAL, RS, and VRS were all primary investigators of this study, contributed to the planning of the study, were involved in the clinical examinations and collection of data. JIG provided insight in the interpretation of the data. CH and AJS assisted with conduction of the study, data analyses, and development of the manuscript. All authors contributed to the writing and critical review of the manuscript and approved the final manuscript.
Declaration of Conflicting Interests
The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Marsha Treadwell received consulting fees and travel and meeting reimbursement from BioMarin.
Paul R. Harmatz received consulting fees from BioMarin, Shire, Genzyme, Alexion, Chiesi, Inventiva, PTC, and ReGenX Bio; he received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
Barbara K. Burton received consulting fees from BioMarin, Shire, Genzyme, Horizon Pharma, Alexion, and ReGenX Bio; she received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, Cytonet, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
John J. Mitchell received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, and Shire.
Nicole Muschol received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Amicus.
Simon A. Jones worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received a honorarium.
Gregory M. Pastores received consulting fees from BioMarin, Genzyme, Pfizer, and Shire.
Heather A. Lau received consulting fees from Genzyme, Pfizer, and BioMarin; she received research funding from Genzyme, NTSAD, BioMarin, Ultragenyx, Pfizer, Shire, Amicus, and GSK and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Pfizer.
Rebecca Sparkes received consulting fees, research funding, and travel and meeting reimbursement from BioMarin.
V. Reid Sutton worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received an honorarium.
Christine Haller is a former employee of BioMarin Pharmaceutical Inc. and a current employee of Ultragenyx.
Bianca Meesen is an employee of Ismar Healthcare NV.
Adam J. Shaywitz is an employee of BioMarin Pharmaceutical Inc. and holder of stock options.
Jeffrey I. Gold received speaker fees and travel reimbursement from BioMarin.
Gregory M. Pastores
University College Dublin, Mater Misericordiae University Hospital, Dublin, IrelandUniversity College DublinIrelandDublin, IrelandUniversity College Dublin, Mater Misericordiae University Hospital, Dublin, Ireland
New York University School of Medicine, New York, NY, USANew York University School of MedicineUSANew York, NY, USANew York University School of Medicine, New York, NY, USA
Authors’ Contributions
MT was consultant to the study, designed the plan for the pain assessment strategies used in the present study, scored and interpreted the pain assessments, and reviewed data analyses. PRH, BKB, JJM, NM, SAJ, GMP, HAL, RS, and VRS were all primary investigators of this study, contributed to the planning of the study, were involved in the clinical examinations and collection of data. JIG provided insight in the interpretation of the data. CH and AJS assisted with conduction of the study, data analyses, and development of the manuscript. All authors contributed to the writing and critical review of the manuscript and approved the final manuscript.
Declaration of Conflicting Interests
The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Marsha Treadwell received consulting fees and travel and meeting reimbursement from BioMarin.
Paul R. Harmatz received consulting fees from BioMarin, Shire, Genzyme, Alexion, Chiesi, Inventiva, PTC, and ReGenX Bio; he received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
Barbara K. Burton received consulting fees from BioMarin, Shire, Genzyme, Horizon Pharma, Alexion, and ReGenX Bio; she received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, Cytonet, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
John J. Mitchell received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, and Shire.
Nicole Muschol received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Amicus.
Simon A. Jones worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received a honorarium.
Gregory M. Pastores received consulting fees from BioMarin, Genzyme, Pfizer, and Shire.
Heather A. Lau received consulting fees from Genzyme, Pfizer, and BioMarin; she received research funding from Genzyme, NTSAD, BioMarin, Ultragenyx, Pfizer, Shire, Amicus, and GSK and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Pfizer.
Rebecca Sparkes received consulting fees, research funding, and travel and meeting reimbursement from BioMarin.
V. Reid Sutton worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received an honorarium.
Christine Haller is a former employee of BioMarin Pharmaceutical Inc. and a current employee of Ultragenyx.
Bianca Meesen is an employee of Ismar Healthcare NV.
Adam J. Shaywitz is an employee of BioMarin Pharmaceutical Inc. and holder of stock options.
Jeffrey I. Gold received speaker fees and travel reimbursement from BioMarin.
Heather A. Lau
New York University School of Medicine, New York, NY, USANew York University School of MedicineUSANew York, NY, USANew York University School of Medicine, New York, NY, USA
Authors’ Contributions
MT was consultant to the study, designed the plan for the pain assessment strategies used in the present study, scored and interpreted the pain assessments, and reviewed data analyses. PRH, BKB, JJM, NM, SAJ, GMP, HAL, RS, and VRS were all primary investigators of this study, contributed to the planning of the study, were involved in the clinical examinations and collection of data. JIG provided insight in the interpretation of the data. CH and AJS assisted with conduction of the study, data analyses, and development of the manuscript. All authors contributed to the writing and critical review of the manuscript and approved the final manuscript.
Declaration of Conflicting Interests
The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Marsha Treadwell received consulting fees and travel and meeting reimbursement from BioMarin.
Paul R. Harmatz received consulting fees from BioMarin, Shire, Genzyme, Alexion, Chiesi, Inventiva, PTC, and ReGenX Bio; he received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
Barbara K. Burton received consulting fees from BioMarin, Shire, Genzyme, Horizon Pharma, Alexion, and ReGenX Bio; she received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, Cytonet, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
John J. Mitchell received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, and Shire.
Nicole Muschol received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Amicus.
Simon A. Jones worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received a honorarium.
Gregory M. Pastores received consulting fees from BioMarin, Genzyme, Pfizer, and Shire.
Heather A. Lau received consulting fees from Genzyme, Pfizer, and BioMarin; she received research funding from Genzyme, NTSAD, BioMarin, Ultragenyx, Pfizer, Shire, Amicus, and GSK and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Pfizer.
Rebecca Sparkes received consulting fees, research funding, and travel and meeting reimbursement from BioMarin.
V. Reid Sutton worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received an honorarium.
Christine Haller is a former employee of BioMarin Pharmaceutical Inc. and a current employee of Ultragenyx.
Bianca Meesen is an employee of Ismar Healthcare NV.
Adam J. Shaywitz is an employee of BioMarin Pharmaceutical Inc. and holder of stock options.
Jeffrey I. Gold received speaker fees and travel reimbursement from BioMarin.
Rebecca Sparkes
Alberta Children’s Hospital, Calgary, Alberta, CanadaAlberta Children’s HospitalCanadaCalgary, Alberta, CanadaAlberta Children’s Hospital, Calgary, Alberta, Canada
Authors’ Contributions
MT was consultant to the study, designed the plan for the pain assessment strategies used in the present study, scored and interpreted the pain assessments, and reviewed data analyses. PRH, BKB, JJM, NM, SAJ, GMP, HAL, RS, and VRS were all primary investigators of this study, contributed to the planning of the study, were involved in the clinical examinations and collection of data. JIG provided insight in the interpretation of the data. CH and AJS assisted with conduction of the study, data analyses, and development of the manuscript. All authors contributed to the writing and critical review of the manuscript and approved the final manuscript.
Declaration of Conflicting Interests
The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Marsha Treadwell received consulting fees and travel and meeting reimbursement from BioMarin.
Paul R. Harmatz received consulting fees from BioMarin, Shire, Genzyme, Alexion, Chiesi, Inventiva, PTC, and ReGenX Bio; he received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
Barbara K. Burton received consulting fees from BioMarin, Shire, Genzyme, Horizon Pharma, Alexion, and ReGenX Bio; she received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, Cytonet, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
John J. Mitchell received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, and Shire.
Nicole Muschol received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Amicus.
Simon A. Jones worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received a honorarium.
Gregory M. Pastores received consulting fees from BioMarin, Genzyme, Pfizer, and Shire.
Heather A. Lau received consulting fees from Genzyme, Pfizer, and BioMarin; she received research funding from Genzyme, NTSAD, BioMarin, Ultragenyx, Pfizer, Shire, Amicus, and GSK and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Pfizer.
Rebecca Sparkes received consulting fees, research funding, and travel and meeting reimbursement from BioMarin.
V. Reid Sutton worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received an honorarium.
Christine Haller is a former employee of BioMarin Pharmaceutical Inc. and a current employee of Ultragenyx.
Bianca Meesen is an employee of Ismar Healthcare NV.
Adam J. Shaywitz is an employee of BioMarin Pharmaceutical Inc. and holder of stock options.
Jeffrey I. Gold received speaker fees and travel reimbursement from BioMarin.
V. Reid Sutton
Baylor College of Medicine & Texas Children’s Hospital, Houston, TX, USABaylor College of MedicineUSAHouston, TX, USABaylor College of Medicine & Texas Children’s Hospital, Houston, TX, USA
Authors’ Contributions
MT was consultant to the study, designed the plan for the pain assessment strategies used in the present study, scored and interpreted the pain assessments, and reviewed data analyses. PRH, BKB, JJM, NM, SAJ, GMP, HAL, RS, and VRS were all primary investigators of this study, contributed to the planning of the study, were involved in the clinical examinations and collection of data. JIG provided insight in the interpretation of the data. CH and AJS assisted with conduction of the study, data analyses, and development of the manuscript. All authors contributed to the writing and critical review of the manuscript and approved the final manuscript.
Declaration of Conflicting Interests
The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Marsha Treadwell received consulting fees and travel and meeting reimbursement from BioMarin.
Paul R. Harmatz received consulting fees from BioMarin, Shire, Genzyme, Alexion, Chiesi, Inventiva, PTC, and ReGenX Bio; he received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
Barbara K. Burton received consulting fees from BioMarin, Shire, Genzyme, Horizon Pharma, Alexion, and ReGenX Bio; she received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, Cytonet, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
John J. Mitchell received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, and Shire.
Nicole Muschol received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Amicus.
Simon A. Jones worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received a honorarium.
Gregory M. Pastores received consulting fees from BioMarin, Genzyme, Pfizer, and Shire.
Heather A. Lau received consulting fees from Genzyme, Pfizer, and BioMarin; she received research funding from Genzyme, NTSAD, BioMarin, Ultragenyx, Pfizer, Shire, Amicus, and GSK and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Pfizer.
Rebecca Sparkes received consulting fees, research funding, and travel and meeting reimbursement from BioMarin.
V. Reid Sutton worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received an honorarium.
Christine Haller is a former employee of BioMarin Pharmaceutical Inc. and a current employee of Ultragenyx.
Bianca Meesen is an employee of Ismar Healthcare NV.
Adam J. Shaywitz is an employee of BioMarin Pharmaceutical Inc. and holder of stock options.
Jeffrey I. Gold received speaker fees and travel reimbursement from BioMarin.
Bianca Meesen
Ismar Healthcare, Lier, BelgiumIsmar HealthcareBelgiumLier, BelgiumIsmar Healthcare, Lier, Belgium
Authors’ Contributions
MT was consultant to the study, designed the plan for the pain assessment strategies used in the present study, scored and interpreted the pain assessments, and reviewed data analyses. PRH, BKB, JJM, NM, SAJ, GMP, HAL, RS, and VRS were all primary investigators of this study, contributed to the planning of the study, were involved in the clinical examinations and collection of data. JIG provided insight in the interpretation of the data. CH and AJS assisted with conduction of the study, data analyses, and development of the manuscript. All authors contributed to the writing and critical review of the manuscript and approved the final manuscript.
Declaration of Conflicting Interests
The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Marsha Treadwell received consulting fees and travel and meeting reimbursement from BioMarin.
Paul R. Harmatz received consulting fees from BioMarin, Shire, Genzyme, Alexion, Chiesi, Inventiva, PTC, and ReGenX Bio; he received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
Barbara K. Burton received consulting fees from BioMarin, Shire, Genzyme, Horizon Pharma, Alexion, and ReGenX Bio; she received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, Cytonet, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
John J. Mitchell received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, and Shire.
Nicole Muschol received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Amicus.
Simon A. Jones worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received a honorarium.
Gregory M. Pastores received consulting fees from BioMarin, Genzyme, Pfizer, and Shire.
Heather A. Lau received consulting fees from Genzyme, Pfizer, and BioMarin; she received research funding from Genzyme, NTSAD, BioMarin, Ultragenyx, Pfizer, Shire, Amicus, and GSK and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Pfizer.
Rebecca Sparkes received consulting fees, research funding, and travel and meeting reimbursement from BioMarin.
V. Reid Sutton worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received an honorarium.
Christine Haller is a former employee of BioMarin Pharmaceutical Inc. and a current employee of Ultragenyx.
Bianca Meesen is an employee of Ismar Healthcare NV.
Adam J. Shaywitz is an employee of BioMarin Pharmaceutical Inc. and holder of stock options.
Jeffrey I. Gold received speaker fees and travel reimbursement from BioMarin.
Christine A. Haller
BioMarin Pharmaceutical Inc., Novato, CA, USABioMarin Pharmaceutical Inc.USANovato, CA, USABioMarin Pharmaceutical Inc., Novato, CA, USA
Authors’ Contributions
MT was consultant to the study, designed the plan for the pain assessment strategies used in the present study, scored and interpreted the pain assessments, and reviewed data analyses. PRH, BKB, JJM, NM, SAJ, GMP, HAL, RS, and VRS were all primary investigators of this study, contributed to the planning of the study, were involved in the clinical examinations and collection of data. JIG provided insight in the interpretation of the data. CH and AJS assisted with conduction of the study, data analyses, and development of the manuscript. All authors contributed to the writing and critical review of the manuscript and approved the final manuscript.
Declaration of Conflicting Interests
The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Marsha Treadwell received consulting fees and travel and meeting reimbursement from BioMarin.
Paul R. Harmatz received consulting fees from BioMarin, Shire, Genzyme, Alexion, Chiesi, Inventiva, PTC, and ReGenX Bio; he received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
Barbara K. Burton received consulting fees from BioMarin, Shire, Genzyme, Horizon Pharma, Alexion, and ReGenX Bio; she received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, Cytonet, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
John J. Mitchell received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, and Shire.
Nicole Muschol received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Amicus.
Simon A. Jones worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received a honorarium.
Gregory M. Pastores received consulting fees from BioMarin, Genzyme, Pfizer, and Shire.
Heather A. Lau received consulting fees from Genzyme, Pfizer, and BioMarin; she received research funding from Genzyme, NTSAD, BioMarin, Ultragenyx, Pfizer, Shire, Amicus, and GSK and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Pfizer.
Rebecca Sparkes received consulting fees, research funding, and travel and meeting reimbursement from BioMarin.
V. Reid Sutton worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received an honorarium.
Christine Haller is a former employee of BioMarin Pharmaceutical Inc. and a current employee of Ultragenyx.
Bianca Meesen is an employee of Ismar Healthcare NV.
Adam J. Shaywitz is an employee of BioMarin Pharmaceutical Inc. and holder of stock options.
Jeffrey I. Gold received speaker fees and travel reimbursement from BioMarin.
Adam J. Shaywitz
BioMarin Pharmaceutical Inc., Novato, CA, USABioMarin Pharmaceutical Inc.USANovato, CA, USABioMarin Pharmaceutical Inc., Novato, CA, USA
Authors’ Contributions
MT was consultant to the study, designed the plan for the pain assessment strategies used in the present study, scored and interpreted the pain assessments, and reviewed data analyses. PRH, BKB, JJM, NM, SAJ, GMP, HAL, RS, and VRS were all primary investigators of this study, contributed to the planning of the study, were involved in the clinical examinations and collection of data. JIG provided insight in the interpretation of the data. CH and AJS assisted with conduction of the study, data analyses, and development of the manuscript. All authors contributed to the writing and critical review of the manuscript and approved the final manuscript.
Declaration of Conflicting Interests
The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Marsha Treadwell received consulting fees and travel and meeting reimbursement from BioMarin.
Paul R. Harmatz received consulting fees from BioMarin, Shire, Genzyme, Alexion, Chiesi, Inventiva, PTC, and ReGenX Bio; he received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
Barbara K. Burton received consulting fees from BioMarin, Shire, Genzyme, Horizon Pharma, Alexion, and ReGenX Bio; she received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, Cytonet, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
John J. Mitchell received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, and Shire.
Nicole Muschol received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Amicus.
Simon A. Jones worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received a honorarium.
Gregory M. Pastores received consulting fees from BioMarin, Genzyme, Pfizer, and Shire.
Heather A. Lau received consulting fees from Genzyme, Pfizer, and BioMarin; she received research funding from Genzyme, NTSAD, BioMarin, Ultragenyx, Pfizer, Shire, Amicus, and GSK and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Pfizer.
Rebecca Sparkes received consulting fees, research funding, and travel and meeting reimbursement from BioMarin.
V. Reid Sutton worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received an honorarium.
Christine Haller is a former employee of BioMarin Pharmaceutical Inc. and a current employee of Ultragenyx.
Bianca Meesen is an employee of Ismar Healthcare NV.
Adam J. Shaywitz is an employee of BioMarin Pharmaceutical Inc. and holder of stock options.
Jeffrey I. Gold received speaker fees and travel reimbursement from BioMarin.
Jeffrey I. Gold
Department of Anesthesiology, Pediatrics, and Psychiatry & Behavioral Sciences, Children’s Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA, USAUniversity of Southern CaliforniaUSALos Angeles, CA, USADepartment of Anesthesiology, Pediatrics, and Psychiatry & Behavioral Sciences, Children’s Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA
Department of Anesthesiology Critical Care Medicine, Children’s Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA, USAUniversity of Southern CaliforniaUSALos Angeles, CA, USADepartment of Anesthesiology Critical Care Medicine, Children’s Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA
Authors’ Contributions
MT was consultant to the study, designed the plan for the pain assessment strategies used in the present study, scored and interpreted the pain assessments, and reviewed data analyses. PRH, BKB, JJM, NM, SAJ, GMP, HAL, RS, and VRS were all primary investigators of this study, contributed to the planning of the study, were involved in the clinical examinations and collection of data. JIG provided insight in the interpretation of the data. CH and AJS assisted with conduction of the study, data analyses, and development of the manuscript. All authors contributed to the writing and critical review of the manuscript and approved the final manuscript.
Declaration of Conflicting Interests
The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: Marsha Treadwell received consulting fees and travel and meeting reimbursement from BioMarin.
Paul R. Harmatz received consulting fees from BioMarin, Shire, Genzyme, Alexion, Chiesi, Inventiva, PTC, and ReGenX Bio; he received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
Barbara K. Burton received consulting fees from BioMarin, Shire, Genzyme, Horizon Pharma, Alexion, and ReGenX Bio; she received research funding from BioMarin, Shire, Ultragenyx, Genzyme, Alexion, Cytonet, and Armagen and travel and meeting reimbursement from BioMarin, Shire, Genzyme, and Alexion.
John J. Mitchell received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, and Shire.
Nicole Muschol received consulting fees, honoraria, and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Amicus.
Simon A. Jones worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received a honorarium.
Gregory M. Pastores received consulting fees from BioMarin, Genzyme, Pfizer, and Shire.
Heather A. Lau received consulting fees from Genzyme, Pfizer, and BioMarin; she received research funding from Genzyme, NTSAD, BioMarin, Ultragenyx, Pfizer, Shire, Amicus, and GSK and travel and meeting reimbursement from BioMarin, Genzyme, Shire, and Pfizer.
Rebecca Sparkes received consulting fees, research funding, and travel and meeting reimbursement from BioMarin.
V. Reid Sutton worked as consultant and study investigator for BioMarin Pharmaceutical Inc. and has received an honorarium.
Christine Haller is a former employee of BioMarin Pharmaceutical Inc. and a current employee of Ultragenyx.
Bianca Meesen is an employee of Ismar Healthcare NV.
Adam J. Shaywitz is an employee of BioMarin Pharmaceutical Inc. and holder of stock options.
Jeffrey I. Gold received speaker fees and travel reimbursement from BioMarin.