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Bone|November 1, 2020
Hip disease in Mucopolysaccharidoses and Mucolipidoses: A review of mechanisms, interventions and future perspectivesEsmee Oussoren, Margreet A E M Wagenmakers, Bianca Link, et al.
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-analysisMark 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-analysisMark 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 NHSMatthew Walton, Nyanar Jasmine Deng, Mark Corbett, et al.
Orphanet Journal of Rare Diseases|September 24, 2024
Redefining the phenotype of alpha-methylacyl-CoA racemase (AMACR) deficiencyFemke C C Klouwer, Stefan D Roosendaal, Carla E M Hollak, et al.
Orphanet Journal of Rare Diseases|June 5, 2026
The impact of cardiovascular risk factors in non-classical Fabry diseaseBram C F Veldman, Laura van Dussen, Mareen R Datema, 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 DiseaseMatthew 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 perspectivesLothar 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.
Orphanet Journal of Rare Diseases|October 22, 2022
Patients' view on gene therapy development for lysosomal storage disorders: a qualitative studyEline C B Eskes, Cathrien R L Beishuizen, Eleonore M Corazolla, et al.
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