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Orphanet Journal of Rare Diseases
|
August 22, 2025
Enzyme replacement therapy for the treatment of late onset Pompe disease: A systematic review and network meta-analysis
Mark Corbett, Chinyereugo Umemneku-Chikere, Sarah Nevitt, et al.
Health Technology Assessment (Winchester, England)
|
February 16, 2026
Enzyme replacement therapy compared with best supportive care for the treatment of Pompe Disease: a systematic review and network meta-analysis
Mark Corbett, Chinyereugo Umemneku-Chikere, Sarah Nevitt, et al.
The European Journal of Health Economics : HEPAC : Health Economics in Prevention and Care
|
November 18, 2025
The cost-effectiveness of enzyme replacement therapies versus best supportive care for treating late onset Pompe disease in the UK NHS
Matthew Walton, Nyanar Jasmine Deng, Mark Corbett, et al.
Journal of the American College of Cardiology
|
June 25, 2011
Exercise-induced left ventricular outflow tract obstruction in symptomatic patients with Anderson-Fabry disease
Margherita Calcagnino, Constantinos O'Mahony, Caroline Coats, et al.
Orphanet Journal of Rare Diseases
|
September 5, 2020
Registries for orphan drugs: generating evidence or marketing tools?
Carla E M Hollak, Sandra Sirrs, Sibren van den Berg, et al.
Pharmacoeconomics
|
November 3, 2025
Re-anchoring the Value of Innovative Therapies in NICE Decision Making When Comparators are Cost Ineffective: A Case Study of Late-Onset Pompe Disease
Matthew Walton, Nyanar J Deng, Mark Corbett, et al.
Frontiers in Endocrinology
|
August 7, 2023
Anticipated effects of burosumab treatment on long-term clinical sequelae in XLH: expert perspectives
Lothar Seefried, Martin Biosse Duplan, Karine Briot, et al.
The European Journal of Health Economics : HEPAC : Health Economics in Prevention and Care
|
February 27, 2024
Assessment of health state utilities associated with adult and pediatric acid sphingomyelinase deficiency (ASMD)
Louis S Matza, Katie D Stewart, Marie Fournier, et al.
Journal of Inherited Metabolic Disease
|
June 19, 2021
Long-term cognitive and psychosocial outcomes in adults with phenylketonuria
Lynne Aitkenhead, Gauri Krishna, Charlotte Ellerton, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
January 9, 2026
Correspondence on "Phenylalanine hydroxylase deficiency diagnosis and management: A 2023 evidence-based clinical guideline of the American College of Medical Genetics and Genomics (ACMG)" by Smith et al
Peter Burgard, Diana Ballhausen, Julia B Hennermann, et al.
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Search research articles
Search
Showing results (21-30 of 55) with videos related to
Sort By:
Page
of 6
Orphanet Journal of Rare Diseases
|
August 22, 2025
Enzyme replacement therapy for the treatment of late onset Pompe disease: A systematic review and network meta-analysis
Mark Corbett, Chinyereugo Umemneku-Chikere, Sarah Nevitt, et al.
Health Technology Assessment (Winchester, England)
|
February 16, 2026
Enzyme replacement therapy compared with best supportive care for the treatment of Pompe Disease: a systematic review and network meta-analysis
Mark Corbett, Chinyereugo Umemneku-Chikere, Sarah Nevitt, et al.
The European Journal of Health Economics : HEPAC : Health Economics in Prevention and Care
|
November 18, 2025
The cost-effectiveness of enzyme replacement therapies versus best supportive care for treating late onset Pompe disease in the UK NHS
Matthew Walton, Nyanar Jasmine Deng, Mark Corbett, et al.
Journal of the American College of Cardiology
|
June 25, 2011
Exercise-induced left ventricular outflow tract obstruction in symptomatic patients with Anderson-Fabry disease
Margherita Calcagnino, Constantinos O'Mahony, Caroline Coats, et al.
Orphanet Journal of Rare Diseases
|
September 5, 2020
Registries for orphan drugs: generating evidence or marketing tools?
Carla E M Hollak, Sandra Sirrs, Sibren van den Berg, et al.
Pharmacoeconomics
|
November 3, 2025
Re-anchoring the Value of Innovative Therapies in NICE Decision Making When Comparators are Cost Ineffective: A Case Study of Late-Onset Pompe Disease
Matthew Walton, Nyanar J Deng, Mark Corbett, et al.
Frontiers in Endocrinology
|
August 7, 2023
Anticipated effects of burosumab treatment on long-term clinical sequelae in XLH: expert perspectives
Lothar Seefried, Martin Biosse Duplan, Karine Briot, et al.
The European Journal of Health Economics : HEPAC : Health Economics in Prevention and Care
|
February 27, 2024
Assessment of health state utilities associated with adult and pediatric acid sphingomyelinase deficiency (ASMD)
Louis S Matza, Katie D Stewart, Marie Fournier, et al.
Journal of Inherited Metabolic Disease
|
June 19, 2021
Long-term cognitive and psychosocial outcomes in adults with phenylketonuria
Lynne Aitkenhead, Gauri Krishna, Charlotte Ellerton, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
January 9, 2026
Correspondence on "Phenylalanine hydroxylase deficiency diagnosis and management: A 2023 evidence-based clinical guideline of the American College of Medical Genetics and Genomics (ACMG)" by Smith et al
Peter Burgard, Diana Ballhausen, Julia B Hennermann, et al.
Page
of 6