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Journal of Health Economics and Outcomes Research
|
February 16, 2026
Trial Interviews to Explore Glycogen Storage Disease Type Ia Patient Experiences Following Gene Therapy
Diane M Turner-Bowker, Jessica Butler, Shayna Egan, et al.
Molecular Genetics and Metabolism
|
July 25, 2018
Safety issues associated with dietary management in patients with hepatic glycogen storage disease
Thomas A H Steunenberg, Fabian Peeks, Irene J Hoogeveen, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
December 11, 2012
Achieving the "triple aim" for inborn errors of metabolism: a review of challenges to outcomes research and presentation of a new practice-based evidence framework
Beth K Potter, Pranesh Chakraborty, Jonathan B Kronick, et al.
JIMD Reports
|
November 22, 2017
Cardiopulmonary Exercise Testing Reflects Improved Exercise Capacity in Response to Treatment in Morquio A Patients: Results of a 52-Week Pilot Study of Two Different Doses of Elosulfase Alfa
Kenneth I Berger, Barbara K Burton, Gregory D Lewis, et al.
Molecular Genetics and Metabolism Reports
|
September 14, 2016
Pregnancy in patients with mucopolysaccharidosis: a case series
Fiona J Stewart, Andrew Bentley, Barbara K Burton, et al.
Molecular Genetics and Metabolism
|
December 18, 2017
Impact of long-term elosulfase alfa on activities of daily living in patients with Morquio A syndrome in an open-label, multi-center, phase 3 extension study
Christian J Hendriksz, Rossella Parini, Moeenaldeen D AlSayed, et al.
Orphanet Journal of Rare Diseases
|
October 31, 2025
Long-term outcomes of elosulfase alfa enzyme replacement therapy in adults with MPS IVA: a sub-analysis of the Morquio A Registry Study (MARS)
Karolina M Stepien, Barbara K Burton, Michael B Bober, et al.
Journal of Inherited Metabolic Disease
|
August 25, 2016
Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome
Christian J Hendriksz, Kenneth I Berger, Rossella Parini, et al.
American Journal of Medical Genetics. Part A
|
June 13, 2015
Safety and physiological effects of two different doses of elosulfase alfa in patients with morquio a syndrome: A randomized, double-blind, pilot study
Barbara K Burton, Kenneth I Berger, Gregory D Lewis, et al.
Molecular Genetics and Metabolism
|
September 10, 2022
Findings from the Morquio A Registry Study (MARS) after 6 years: Long-term outcomes of MPS IVA patients treated with elosulfase alfa
John J Mitchell, Barbara K Burton, Michael B Bober, et al.
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Search research articles
Search
Showing results (31-40 of 73) with videos related to
Sort By:
Page
of 8
Journal of Health Economics and Outcomes Research
|
February 16, 2026
Trial Interviews to Explore Glycogen Storage Disease Type Ia Patient Experiences Following Gene Therapy
Diane M Turner-Bowker, Jessica Butler, Shayna Egan, et al.
Molecular Genetics and Metabolism
|
July 25, 2018
Safety issues associated with dietary management in patients with hepatic glycogen storage disease
Thomas A H Steunenberg, Fabian Peeks, Irene J Hoogeveen, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
December 11, 2012
Achieving the "triple aim" for inborn errors of metabolism: a review of challenges to outcomes research and presentation of a new practice-based evidence framework
Beth K Potter, Pranesh Chakraborty, Jonathan B Kronick, et al.
JIMD Reports
|
November 22, 2017
Cardiopulmonary Exercise Testing Reflects Improved Exercise Capacity in Response to Treatment in Morquio A Patients: Results of a 52-Week Pilot Study of Two Different Doses of Elosulfase Alfa
Kenneth I Berger, Barbara K Burton, Gregory D Lewis, et al.
Molecular Genetics and Metabolism Reports
|
September 14, 2016
Pregnancy in patients with mucopolysaccharidosis: a case series
Fiona J Stewart, Andrew Bentley, Barbara K Burton, et al.
Molecular Genetics and Metabolism
|
December 18, 2017
Impact of long-term elosulfase alfa on activities of daily living in patients with Morquio A syndrome in an open-label, multi-center, phase 3 extension study
Christian J Hendriksz, Rossella Parini, Moeenaldeen D AlSayed, et al.
Orphanet Journal of Rare Diseases
|
October 31, 2025
Long-term outcomes of elosulfase alfa enzyme replacement therapy in adults with MPS IVA: a sub-analysis of the Morquio A Registry Study (MARS)
Karolina M Stepien, Barbara K Burton, Michael B Bober, et al.
Journal of Inherited Metabolic Disease
|
August 25, 2016
Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome
Christian J Hendriksz, Kenneth I Berger, Rossella Parini, et al.
American Journal of Medical Genetics. Part A
|
June 13, 2015
Safety and physiological effects of two different doses of elosulfase alfa in patients with morquio a syndrome: A randomized, double-blind, pilot study
Barbara K Burton, Kenneth I Berger, Gregory D Lewis, et al.
Molecular Genetics and Metabolism
|
September 10, 2022
Findings from the Morquio A Registry Study (MARS) after 6 years: Long-term outcomes of MPS IVA patients treated with elosulfase alfa
John J Mitchell, Barbara K Burton, Michael B Bober, et al.
Page
of 8