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Orphanet Journal of Rare Diseases
|
August 29, 2023
A systematic review of clinical effectiveness and safety for historical and current treatment options for metachromatic leukodystrophy in children, including atidarsagene autotemcel
Nigel Armstrong, Andrew Olaye, Caro Noake, et al.
Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research
|
May 14, 2018
Economic Modeling Considerations for Rare Diseases
Isobel Pearson, Ben Rothwell, Andrew Olaye, et al.
Advanced Drug Delivery Reviews
|
June 2, 2007
Polymer carriers for drug delivery in tissue engineering
Marina Sokolsky-Papkov, Kapil Agashi, Andrew Olaye, et al.
Expert Review of Pharmacoeconomics & Outcomes Research
|
December 8, 2021
Evaluating discrete choice experiment willingness to pay [DCE-WTP] analysis and relative social willingness to pay [RS-WTP] analysis in a health technology assessment of a treatment for an ultra-rare childhood disease [CLN2]
Domenico Moro, Michael Schlander, Harry Telser, et al.
International Journal of Neonatal Screening
|
July 25, 2024
Exploring the Cost-Effectiveness of Newborn Screening for Metachromatic Leukodystrophy (MLD) in the UK
Karen Bean, Simon A Jones, Anupam Chakrapani, et al.
Plos One
|
May 22, 2024
Exploring concurrent validity of the CLN2 Clinical Rating Scale: Comparison to PedsQL using cerliponase alfa clinical trial data
Nicola Specchio, Paul Gissen, Emily de Los Reyes, et al.
Orphanet Journal of Rare Diseases
|
November 19, 2014
A systematic review of the prevalence of Morquio A syndrome: challenges for study reporting in rare diseases
Regina M Leadley, Shona Lang, Kate Misso, et al.
Orphanet Journal of Rare Diseases
|
May 13, 2021
Investigating health-related quality of life in rare diseases: a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2)
Paul Gissen, Nicola Specchio, Andrew Olaye, et al.
Page
of 1
Search research articles
Search
Showing results (1-10 of 8) with videos related to
Sort By:
Page
of 1
Orphanet Journal of Rare Diseases
|
August 29, 2023
A systematic review of clinical effectiveness and safety for historical and current treatment options for metachromatic leukodystrophy in children, including atidarsagene autotemcel
Nigel Armstrong, Andrew Olaye, Caro Noake, et al.
Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research
|
May 14, 2018
Economic Modeling Considerations for Rare Diseases
Isobel Pearson, Ben Rothwell, Andrew Olaye, et al.
Advanced Drug Delivery Reviews
|
June 2, 2007
Polymer carriers for drug delivery in tissue engineering
Marina Sokolsky-Papkov, Kapil Agashi, Andrew Olaye, et al.
Expert Review of Pharmacoeconomics & Outcomes Research
|
December 8, 2021
Evaluating discrete choice experiment willingness to pay [DCE-WTP] analysis and relative social willingness to pay [RS-WTP] analysis in a health technology assessment of a treatment for an ultra-rare childhood disease [CLN2]
Domenico Moro, Michael Schlander, Harry Telser, et al.
International Journal of Neonatal Screening
|
July 25, 2024
Exploring the Cost-Effectiveness of Newborn Screening for Metachromatic Leukodystrophy (MLD) in the UK
Karen Bean, Simon A Jones, Anupam Chakrapani, et al.
Plos One
|
May 22, 2024
Exploring concurrent validity of the CLN2 Clinical Rating Scale: Comparison to PedsQL using cerliponase alfa clinical trial data
Nicola Specchio, Paul Gissen, Emily de Los Reyes, et al.
Orphanet Journal of Rare Diseases
|
November 19, 2014
A systematic review of the prevalence of Morquio A syndrome: challenges for study reporting in rare diseases
Regina M Leadley, Shona Lang, Kate Misso, et al.
Orphanet Journal of Rare Diseases
|
May 13, 2021
Investigating health-related quality of life in rare diseases: a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2)
Paul Gissen, Nicola Specchio, Andrew Olaye, et al.
Page
of 1