Search Results - "Olaye, Andrew"

A systematic review of clinical effectiveness and safety for historical and current treatment options for metachromatic leukodystrophy in children, including atidarsagene autotemcel by Armstrong, Nigel, Olaye, Andrew, Noake, Caro, Pang, Francis

“…To understand the benefit-risk profile for historical and current treatments for MLD. A systematic review was conducted on the effectiveness, safety, and costs…”
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Exploring concurrent validity of the CLN2 Clinical Rating Scale: Comparison to PedsQL using cerliponase alfa clinical trial data by Specchio, Nicola, Gissen, Paul, de Los Reyes, Emily, Olaye, Andrew, Camp, Charlotte, Curteis, Tristan, Griffiths, Annabel, Butt, Thomas, Cohen-Pfeffer, Jessica, Slasor, Peter, Sisic, Zlatko, Jain, Mohit, Schulz, Angela

“…The CLN2 Clinical Rating Scale evaluates disease progression in CLN2 disease, an ultra-rare, neurodegenerative disorder with late infantile onset. To validate…”
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A systematic review of the prevalence of Morquio A syndrome: challenges for study reporting in rare diseases by Leadley, Regina M, Lang, Shona, Misso, Kate, Bekkering, Trudy, Ross, Janine, Akiyama, Takeyuki, Fietz, Michael, Giugliani, Roberto, Hendriksz, Chris J, Hock, Ngu Lock, McGill, Jim, Olaye, Andrew, Jain, Mohit, Kleijnen, Jos

“…Morquio A (MPS IVA) is a rare disease characterised by a deficiency of N-acetylgalactosamine-6 sulfatase (GALNS) and presenting with short stature, abnormal…”
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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) by Gissen, Paul, Specchio, Nicola, Olaye, Andrew, Jain, Mohit, Butt, Thomas, Ghosh, Wrik, Ruban-Fell, Benjamin, Griffiths, Annabel, Camp, Charlotte, Sisic, Zlatko, Schwering, Christoph, Wibbeler, Eva, Trivisano, Marina, Lee, Laura, Nickel, Miriam, Mortensen, Amanda, Schulz, Angela

“…Utility studies enable preference-based quantification of a disease's impact on patients' health-related quality of life (HRQoL). It is often difficult to…”
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Exploring the Cost-Effectiveness of Newborn Screening for Metachromatic Leukodystrophy (MLD) in the UK by Bean, Karen, Jones, Simon A, Chakrapani, Anupam, Vijay, Suresh, Wu, Teresa, Church, Heather, Chanson, Charlotte, Olaye, Andrew, Miller, Beckley, Jensen, Ivar, Pang, Francis

“…Metachromatic leukodystrophy (MLD) is a fatal inherited lysosomal storage disease that can be detected through newborn bloodspot screening. The feasibility of…”
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The Challenges of Living with and Caring for a Child or Children Affected by Neuronal Ceroid Lipofuscinosis Type 2 Disease: In-Depth Family Surveys in the United Kingdom and Germany by Schulz, Angela, Jain, Mohit, Butt, Thomas, Ballinger, Rachel, Eliasson, Lina, Macey, Jake, Peasgood, Tessa, Olaye, Andrew, Terzakis-Snyder, Irini-Alexia, Dyck, Iris, West, Andrea

“…Abstract Limited research has investigated the challenges faced by families caring for children with neuronal ceroid lipofuscinosis type 2 (CLN2) disease…”
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Relationship Between Patient-Reported Outcomes and Clinical Outcomes in Patients With Morquio A Syndrome by Lampe, Christina, Jain, Mohit, Olaye, Andrew, Meesen, Bianca, Decker, Celeste, Mengel, Eugen

“…This cross-sectional analysis assessed the correlation between patient-reported outcomes (PROs) and clinical outcomes in 24 German patients with Morquio A…”
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Economic Modeling Considerations for Rare Diseases by Pearson, Isobel, Rothwell, Ben, Olaye, Andrew, Knight, Christopher

“…To identify challenges that affect the feasibility and rigor of economic models in rare diseases and strategies that manufacturers have employed in health…”
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Polymer carriers for drug delivery in tissue engineering by Sokolsky-Papkov, Marina, Agashi, Kapil, Olaye, Andrew, Shakesheff, Kevin, Domb, Abraham J.

“…Growing demand for tissues and organs for transplantation and the inability to meet this need using by autogeneic (from the host) or allogeneic (from the same…”
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Health-related quality of life in metachromatic leukodystrophy based on a societal utility study in the UK by Pang, Francis, DeFreitas, Hayley, Lloyd, Andrew, Olaye, Andrew, Nafees, Beenish

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The cost-effectiveness of atidarsagene autotemcel for the treatment of metachromatic leukodystrophy in France by Pang, Francis, Dean, Rebecca, Jensen, Ivar, Tehard, Bertrand, Roze, Stephane, Olaye, Andrew, Bean, Karen, Miller, Beck

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Demographic and clinical characteristics of patients with metachromatic leukodystrophy in the United Kingdom: Interim results from an observational real-world study by Jones, Simon, Davison, James, Mooney, Peter, Campbell, Laura, Baldock, Laura, Wallington, Mike, Olaye, Andrew E., Chakrapani, Anupam

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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] by Moro, Domenico, Schlander, Michael, Telser, Harry, Sola-Morales, Oriol, Clark, Michael David, Olaye, Andrew, Camp, Charlotte, Jain, Mohit, Butt, Thomas, Bakshi, Sumeet

“…Conventional cost-effectiveness analysis [CEA] using cost per QALY thresholds may counteract other incentives introduced to foster development of treatments…”
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Polymer carriers for drug delivery in tissue engineering: Matrices and scaffolds for drug delivery in tissue engineering by SOKOLSKY-PAPKOV, Marina, AGASHI, Kapil, OLAYE, Andrew, SHAKESHEFF, Kevin, DOMB, Abraham J

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Differentiation of embryonic stem cells through controlled release of growth factors from microspheres by Olaye, Eghosa Omoregie Andrew

“…The development of microspheres for the sustained delivery of protein and small drug delivery has been utilised in tissue engineering and drug delivery…”
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