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ArticlesPeer ReviewedFull Text
New Developments in the Treatment of X-Linked Hypophosphataemia: Implications for Clinical Management
Author / Creator:Saraff, Vrinda ; Nadar, Ruchi ; Högler, Wolfgang
Publisher:Cham: Springer International Publishing
Journal title:Paediatric drugs, 2020-04, Vol.22 (2), p.113-121
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A patient-centred and multi-stakeholder co-designed observational prospective study protocol: Example of the adolescent experience of treatment for X-linked hypophosphataemia (XLH)
Author / Creator:Saraff, Vrinda ; Boot, Annemieke M ; Linglart, Agnès ; Semler, Oliver ; Harvengt, Pol ; Williams, Angela ; Bailey, Karen M A ; Glen, Fiona ; Davies, Elin Haf ; Wood, Sue ; Greentree, Stephen ; Rylands, Angela J
Publisher:United States: Public Library of Science
Journal title:PloS one, 2024-01, Vol.19 (1), p.e0295080-e0295080
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Infigratinib in children with achondroplasia: the PROPEL and PROPEL 2 studies
Author / Creator:Savarirayan, Ravi ; De Bergua, Josep Maria ; Arundel, Paul ; McDevitt, Helen ; Cormier-Daire, Valerie ; Saraff, Vrinda ; Skae, Mars ; Delgado, Borja ; Leiva-Gea, Antonio ; Santos-Simarro, Fernando ; Salles, Jean Pierre ; Nicolino, Marc ; Rossi, Massimiliano ; Kannu, Peter ; Bober, Michael B. ; Phillips, John ; Saal, Howard ; Harmatz, Paul ; Burren, Christine ; Gotway, Garrett ; Cho, Terry ; Muslimova, Elena ; Weng, Richard ; Rogoff, Daniela ; Hoover-Fong, Julie ; Irving, Melita
Publisher:London, England: SAGE Publications
Journal title:Therapeutic advances in musculoskeletal disease, 2022-03, Vol.14, p.1759720X221084848-1759720X221084848
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Cardiac, bone and growth plate manifestations in hypocalcemic infants: revealing the hidden body of the vitamin D deficiency iceberg
Author / Creator:Uday, Suma ; Fratzl-Zelman, Nadja ; Roschger, Paul ; Klaushofer, Klaus ; Chikermane, Ashish ; Saraff, Vrinda ; Tulchinsky, Ted ; Thacher, Tom D ; Marton, Tamas ; Högler, Wolfgang
Publisher:England: BioMed Central Ltd
Journal title:BMC pediatrics, 2018-06, Vol.18 (1), p.183-183, Article 183
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Bi‐allelic mutation in SEC16B alters collagen trafficking and increases ER stress
Author / Creator:El‐Gazzar, Ahmed ; Voraberger, Barbara ; Rauch, Frank ; Mairhofer, Mario ; Schmidt, Katy ; Guillemyn, Brecht ; Mitulović, Goran ; Reiterer, Veronika ; Haun, Margot ; Mayr, Michaela M ; Mayr, Johannes A ; Kimeswenger, Susanne ; Drews, Oliver ; Saraff, Vrinda ; Shaw, Nick ; Fratzl‐Zelman, Nadja ; Symoens, Sofie ; Farhan, Hesso ; Högler, Wolfgang
Publisher:London: Nature Publishing Group UK
Journal title:EMBO molecular medicine, 2023-04, Vol.15 (4), p.e16834-n/a
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Phenotypic Spectrum in Osteogenesis Imperfecta Due to Mutations in TMEM38B: Unraveling a Complex Cellular Defect
Author / Creator:Webb, Emma A. ; Balasubramanian, Meena ; Fratzl-Zelman, Nadja ; Cabral, Wayne A. ; Titheradge, Hannah ; Alsaedi, Atif ; Saraff, Vrinda ; Vogt, Julie ; Cole, Trevor ; Stewart, Susan ; Crabtree, Nicola J. ; Sargent, Brandi M. ; Gamsjaeger, Sonja ; Paschalis, Eleftherios P. ; Roschger, Paul ; Klaushofer, Klaus ; Shaw, Nick J. ; Marini, Joan C. ; Högler, Wolfgang
Publisher:Washington, DC: Endocrine Society
Journal title:The journal of clinical endocrinology and metabolism, 2017-06, Vol.102 (6), p.2019-2028
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Healthcare resource utilization in the management of hypophosphatasia in three patients displaying a spectrum of manifestations
Author / Creator:Daniel, Anjali B ; Saraff, Vrinda ; Shaw, Nick J ; Yates, Robert ; Mughal, M Zulf ; Padidela, Raja
Publisher:England: BioMed Central Ltd
Journal title:Orphanet journal of rare diseases, 2018-08, Vol.13 (1), p.142-142, Article 142
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Hypophosphatasia mimicking hypoxic–ischaemic encephalopathy: early recognition and management
Author / Creator:Picton, Aled ; Nadar, Ruchi ; Pelivan, Alexandra ; Garikapati, Vidya ; Saraff, Vrinda
Publisher:England: BMJ Publishing Group Ltd and Royal College of Paediatrics and Child Health
Journal title:Archives of disease in childhood, 2021-02, Vol.106 (2), p.189-191
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Growth hormone prescribing patterns in the UK, 2013–2016
Author / Creator:Shepherd, Sheila ; Saraff, Vrinda ; Shaw, Nick ; Banerjee, Indraneel ; Patel, Leena
Publisher:England: BMJ Publishing Group LTD
Journal title:Archives of disease in childhood, 2019-06, Vol.104 (6), p.583-587
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Non-collagen pathogenic variants resulting in the osteogenesis imperfecta phenotype in children: a single-country observational cohort study
Author / Creator:Thornley, Patrick ; Bishop, Nicholas ; Baker, Duncan ; Brock, Joanna ; Arundel, Paul ; Burren, Christine ; Smithson, Sarah ; DeVile, Catherine ; Crowe, Belinda ; Allgrove, Jeremy ; Saraff, Vrinda ; Shaw, Nick ; Balasubramanian, Meena
Publisher:England: BMJ Publishing Group Ltd and Royal College of Paediatrics and Child Health
Journal title:Archives of disease in childhood, 2022-05, Vol.107 (5), p.486-490
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Efficacy and treatment costs of zoledronate versus pamidronate in paediatric osteoporosis
Author / Creator:Saraff, Vrinda ; Sahota, Jaskiran ; Crabtree, Nicola ; Sakka, Sophia ; Shaw, Nicholas J ; Högler, Wolfgang
Publisher:England: BMJ Publishing Group Ltd
Journal title:Archives of disease in childhood, 2018-01, Vol.103 (1), p.92-94
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Bone Structural Characteristics and Response to Bisphosphonate Treatment in Children With Hajdu-Cheney Syndrome
Author / Creator:Sakka, Sophia ; Gafni, Rachel I ; Davies, Justin H ; Clarke, Bart ; Tebben, Peter ; Samuels, Mark ; Saraff, Vrinda ; Klaushofer, Klaus ; Fratzl-Zelman, Nadja ; Roschger, Paul ; Rauch, Frank ; Högler, Wolfgang
Publisher:Washington, DC: Endocrine Society
Journal title:The journal of clinical endocrinology and metabolism, 2017-11, Vol.102 (11), p.4163-4172
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Sex-, age-, and height-specific reference curves for the 6-min walk test in healthy children and adolescents
Author / Creator:Saraff, Vrinda ; Schneider, Johanna ; Colleselli, Valeria ; Ruepp, Monika ; Rauchenzauner, Markus ; Neururer, Sabrina ; Geiger, Ralf ; Högler, Wolfgang
Publisher:Berlin/Heidelberg: Springer Berlin Heidelberg
Journal title:European journal of pediatrics, 2015-06, Vol.174 (6), p.837-840
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The Growth Hormone Deficiency
Author / Creator:Brettell, Elizabeth ; Högler, Wolfgang ; Woolley, Rebecca ; Cummins, Carole ; Mathers, Jonathan ; Oppong, Raymond ; Roy, Laura ; Khan, Adam ; Hunt, Charmaine ; Dattani, Mehul ; Ong, Ken ; Donaldson, Malcolm ; Harris, Victoria ; Maghnie, Mohamad ; Gregory, John ; Auguste, Peter ; Binder, Gerhard ; Gambol, Carrol ; Dhamaraj, Poonam ; Gevers, Evelien ; Saraff, Vrinda ; Clayton, Peter ; Randell, Tabitha ; Mushtaq, Talat ; Cheetham, Timothy ; Davies, Justin ; Abid, Noina ; Khairi, Ranna El ; Kapelari, Klaus ; Gottardi-Butturini, Elena ; Reiterer-Fröhlich, Elke ; Bonfig, Walter
Publisher:BioMed Central Ltd
Journal title:Trials, 2023-08, Vol.24 (1)
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The Growth Hormone Deficiency (GHD) Reversal Trial: effect on final height of discontinuation versus continuation of growth hormone treatment in pubertal children with isolated GHD—a non-inferiority R...
Author / Creator:Brettell, Elizabeth ; Högler, Wolfgang ; Woolley, Rebecca ; Cummins, Carole ; Mathers, Jonathan ; Oppong, Raymond ; Roy, Laura ; Khan, Adam ; Hunt, Charmaine ; Dattani, Mehul ; Ong, Ken ; Donaldson, Malcolm ; Harris, Victoria ; Maghnie, Mohamad ; Gregory, John ; Auguste, Peter ; Binder, Gerhard ; Gambol, Carrol ; Dhamaraj, Poonam ; Gevers, Evelien ; Saraff, Vrinda ; Clayton, Peter ; Randell, Tabitha ; Mushtaq, Talat ; Cheetham, Timothy ; Davies, Justin ; Abid, Noina ; Khairi, Ranna El ; Kapelari, Klaus ; Gottardi-Butturini, Elena ; Reiterer-Fröhlich, Elke ; Bonfig, Walter
Publisher:London: BioMed Central
Journal title:Current controlled trials in cardiovascular medicine, 2023-08, Vol.24 (1), p.548-548, Article 548
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OR27-03 Oral Infigratinib Treatment Is Well Tolerated And Significantly Increases Height Velocity In Children With Achondroplasia: Month 6 Results From The PROPEL 2 Dose-finding Study
Author / Creator:Savarirayan, Ravi ; De Bergua, Josep Maria ; Arundel, Paul ; Salles, Jean Pierre ; Saraff, Vrinda ; Delgado, Borja ; Leiva-Gea, Antonio ; McDevitt, Helen ; Nicolino, Marc P ; Rossi, Massimiliano ; Salcedo, Maria ; Cormier-Daire, Valerie ; Skae, Mars ; Kannu, Peter ; Bober, Michael B ; Phillips, John ; Saal, Howard ; Harmatz, Paul ; Burren, Christine Pamela ; Candler, Toby ; Cho, Terry ; Muslimova, Elena ; Weng, Richard ; Raj, Supriya ; Hoover-Fong, Julie ; Irving, Melita ; Rogoff, Daniela
Publisher:US: Oxford University Press
Journal title:Journal of the Endocrine Society, 2023-10, Vol.7 (Supplement_1)
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THU165 PROPEL, PROPEL 2 And PROPEL OLE Studies Of Infigratinib In Children With Achondroplasia: Design And Status Of 3 Ongoing Trials
Author / Creator:Savarirayan, Ravi ; De Bergua, Josep Maria ; Arundel, Paul ; McDevitt, Helen ; Cormier-Daire, Valerie ; Saraff, Vrinda ; Skae, Mars ; Delgado, Borja ; Leiva-Gea, Antonio ; Salcedo, Maria ; Salles, Jean-Pierre ; Nicolino, Marc P ; Rossi, Massimiliano ; Kannu, Peter ; Bober, Michael B ; Phillips, John ; Saal, Howard ; Harmatz, Paul ; Burren, Christine ; Candler, Toby ; Cho, Terry ; Muslimova, Elena ; Weng, Richard ; Rogoff, Daniela ; Hoover-Fong, Julie ; Irving, Melita
Publisher:US: Oxford University Press
Journal title:Journal of the Endocrine Society, 2023-10, Vol.7 (Supplement_1)
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THU181 Evaluation Of Bone Mineral Density In A Cohort Of Children With ACH Participating In The PROPEL 2 Study Of Infigratinib
Author / Creator:Savarirayan, Ravi ; De Bergua, Josep Maria ; Arundel, Paul ; Salles, Jean Pierre ; Leiva-Gea, Antonio ; Irving, Melita ; Saraff, Vrinda ; McDevitt, Helen ; Salcedo, Maria ; Nicolino, Marc P ; Cormier-Daire, Valerie ; Kannu, Peter ; Skae, Mars ; Bober, Michael B ; Phillips III, John ; Candler, Toby ; Harmatz, Paul ; Saal, Howard ; Hoover-Fong, Julie ; Muslimova, Elena ; Cho, Terry ; Weng, Richard ; Rogoff, Daniela
Publisher:US: Oxford University Press
Journal title:Journal of the Endocrine Society, 2023-10, Vol.7 (Supplement_1)
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ReviewsPeer ReviewedFull Text
Sunshine and vitamin D
Author / Creator:Saraff, Vrinda ; Shaw, Nick
Publisher:England: BMJ Publishing Group Ltd
Journal title:Archives of disease in childhood, 2016, Vol.101 (2), p.190-192
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ArticlesPeer ReviewedFull Text
RF26 | PMON326 Medical History of Children Enrolled in PROPEL: A Prospective Clinical Assessment Study in Children with Achondroplasia
Author / Creator:Irving, Melita ; De Bergua, Josep Maria ; Rogoff, Daniela ; Savarirayan, Ravi ; Arundel, Paul ; Pierre Salles, Jean ; Leiva-Gea, Antonio ; Saraff, Vrinda ; McDevitt, Helen ; Santos-Simarro, Fernando ; Nicolino, Marc ; Cormier-Daire, Valerie ; Kannu, Peter ; Skae, Mars ; Bober, Michael B ; Phillips III, John ; Burren, Christine ; Harmatz, Paul ; Saal, Howard ; Hoover-Fong, Julie ; Weng, Richard ; Muslimova, Elena ; Cho, Terry
Publisher:Oxford: Oxford University Press
Journal title:Journal of the Endocrine Society, 2022-11, Vol.6 (Supplement_1), p.A644-A644
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New Developments in the Treatment of X-Linked Hypophosphataemia: Implications for Clinical Management

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A patient-centred and multi-stakeholder co-designed observational prospective study protocol: Example of the adolescent experience of treatment for X-linked hypophosphataemia (XLH)

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Infigratinib in children with achondroplasia: the PROPEL and PROPEL 2 studies

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Cardiac, bone and growth plate manifestations in hypocalcemic infants: revealing the hidden body of the vitamin D deficiency iceberg

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Bi‐allelic mutation in SEC16B alters collagen trafficking and increases ER stress

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Phenotypic Spectrum in Osteogenesis Imperfecta Due to Mutations in TMEM38B: Unraveling a Complex Cellular Defect

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Healthcare resource utilization in the management of hypophosphatasia in three patients displaying a spectrum of manifestations

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Hypophosphatasia mimicking hypoxic–ischaemic encephalopathy: early recognition and management

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Growth hormone prescribing patterns in the UK, 2013–2016

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Non-collagen pathogenic variants resulting in the osteogenesis imperfecta phenotype in children: a single-country observational cohort study

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Efficacy and treatment costs of zoledronate versus pamidronate in paediatric osteoporosis

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Bone Structural Characteristics and Response to Bisphosphonate Treatment in Children With Hajdu-Cheney Syndrome

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Sex-, age-, and height-specific reference curves for the 6-min walk test in healthy children and adolescents

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The Growth Hormone Deficiency

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The Growth Hormone Deficiency (GHD) Reversal Trial: effect on final height of discontinuation versus continuation of growth hormone treatment in pubertal children with isolated GHD—a non-inferiority R...

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OR27-03 Oral Infigratinib Treatment Is Well Tolerated And Significantly Increases Height Velocity In Children With Achondroplasia: Month 6 Results From The PROPEL 2 Dose-finding Study

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THU165 PROPEL, PROPEL 2 And PROPEL OLE Studies Of Infigratinib In Children With Achondroplasia: Design And Status Of 3 Ongoing Trials

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THU181 Evaluation Of Bone Mineral Density In A Cohort Of Children With ACH Participating In The PROPEL 2 Study Of Infigratinib

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Sunshine and vitamin D

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RF26 | PMON326 Medical History of Children Enrolled in PROPEL: A Prospective Clinical Assessment Study in Children with Achondroplasia

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