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Persistent growth-promoting effects of vosoritide in children with achondroplasia are accompanied by improvements in physical and social aspects of health-related quality of life Savarirayan, Ravi; Irving, Melita; Wilcox, William R.; Bacino, Carlos A.; Hoover-Fong, Julie E.; Harmatz, Paul; Polgreen, Lynda E.; Mohnike, Klaus; Prada, Carlos E.; Kubota, Takuo; Arundel, Paul; Leiva-Gea, Antonio; Rowell, Richard; Low, Andrea; Sabir, Ian; Huntsman-Labed, Alice; Day, Jonathan 分享 收藏
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Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial Savarirayan, Ravi; Wilcox, William R.; Harmatz, Paul; Phillips, John; Polgreen, Lynda E.; Tofts, Louise; Ozono, Keiichi; Arundel, Paul; Irving, Melita; Bacino, Carlos A.; Basel, Donald; Bober, Michael B.; Charrow, Joel; Mochizuki, Hiroshi; Kotani, Yumiko; Saal, Howard M.; Army, Clare; Jeha, George; Qi, Yulan; Han, Lynn; Fisheleva, Elena; Huntsman-Labed, Alice; Day, Jonathan 分享 收藏
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European Achondroplasia Forum guiding principles for the detection and management of foramen magnum stenosis Irving, Melita; AlSayed, Moeenaldeen; Arundel, Paul; Baujat, Genevieve; Ben-Omran, Tawfeg; Boero, Silvio; Cormier-Daire, Valerie; Fredwall, Svein; Guillen-Navarro, Encarna; Hoyer-Kuhn, Heike; Kunkel, Philip; Lampe, Christian; Maghnie, Mohamad; Mohnike, Klaus; Mortier, Geert; Sousa, Sergio B. 分享 收藏
Growth parameters in children with achondroplasia: A 7-year, prospective, multinational, observational study 软骨发育不全儿童的生长参数: 一项为期7年的前瞻性多国观察性研究 Savarirayan, Ravi; Irving, Melita; Harmatz, Paul; Delgado, Borja; Wilcox, William R.; Philips, John; Owen, Natalie; Bacino, Carlos A.; Tofts, Louise; Charrow, Joel; Polgreen, Lynda E.; Hoover-Fong, Julie; Arundel, Paul; Ginebreda, Ignacio; Saal, Howard M.; Basel, Donald; Font, Rosendo Ullot; Ozono, Keiichi; Bober, Michael B.; Cormier-Daire, Valerie; Kim-Hanh Le Quan Sang; Baujat, Genevieve; Alanay, Yasemin; Rutsch, Frank; Hoernschemeyer, Daniel; Mohnike, Klaus; Mochizuki, Hiroshi; Tajima, Asako; Kotani, Yumiko; Weaver, David D.; White, Klane K.; Army, Clare; Larrimore, Kevin; Gregg, Keith; Jeha, George; Milligan, Claire; Fisheleva, Elena; Huntsman-Labed, Alice; Day, Jonathan 分享 收藏
Associations between height and Health-Related Quality of Life (HRQoL) and functional independence in children with achondroplasia Irving, Melita; Savarirayan, Ravi; Arundel, Paul; Polgreen, Lynda; Mohnike, Klaus; Ozono, Keiichi; Saunders, Michael; Fisheleva, Elena; Huntsman-Labed, Alice; Day, Jonathan 分享 收藏
Monitoring Skull Base Abnormalities in Children with Osteogenesis Imperfecta - Review of Current Practice and a Suggested Clinical Pathway Wadanamby, S.; El Garwany, S.; Connolly, D. J. A.; Arundel, P.; Bishop, N. J.; DeVile, C. J.; Calder, A. D.; Crowe, B.; Burren, C. P.; Saraff, V; Offiah, A. C. 分享 收藏
Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study Savarirayan, Ravi; Tofts, Louise; Irving, Melita; Wilcox, William R.; Bacino, Carlos A.; Hoover-Fong, Julie; Font, Rosendo Ullot; Harmatz, Paul; Rutsch, Frank; Bober, Michael B.; Polgreen, Lynda E.; Ginebreda, Ignacio; Mohnike, Klaus; Charrow, Joel; Hoernschemeyer, Daniel; Ozono, Keiichi; Alanay, Yasemin; Arundel, Paul; Kotani, Yumiko; Yasui, Natsuo; White, Klane K.; Saal, Howard M.; Leiva-Gea, Antonio; Luna-Gonzalez, Felipe; Mochizuki, Hiroshi; Basel, Donald; Porco, Dania M.; Jayaram, Kala; Fisheleva, Elena; Huntsman-Labed, Alice; Day, Jonathan R. S. 分享 收藏
Expanding the phenotype of SPARC-related osteogenesis imperfecta: clinical findings in two patients with pathogenic variants in SPARC and literature review Durkin, Anna; DeVile, Catherine; Arundel, Paul; Bull, Mary; Walsh, Jennifer; Bishop, Nicholas J.; Hupin, Emilie; Parekh, Susan; Nadarajah, Ramesh; Offiah, Amaka C.; Calder, Alistair; Brock, Joanna; Baker, Duncan; Balasubramanian, Meena 分享 收藏
Best practice management guidelines for fibrous dysplasia/McCune-Albright syndrome: a consensus statement from the FD/MAS international consortium (vol 14, 139, 2019) Javaid, Muhammad Kassim; Boyce, Alison; Appelman-Dijkstra, Natasha; Ong, Juling; Defabianis, Patrizia; Offiah, Amaka; Arundel, Paul; Shaw, Nick; Dal Pos, Valter; Underhil, Ann; Portero, Deanna; Heral, Lisa; Heegaard, Anne-Marie; Masi, Laura; Monsell, Fergal; Stanton, Robert; Dijkstra, Pieter Durk Sander; Brandi, Maria Luisa; Chapurlat, Roland; Hamdy, Neveen Agnes Therese; Collins, Michael Terrence 分享 收藏
Elemental formula associated hypophosphataemic rickets 元素配方相关的低磷血症性佝偻病 Uday, S.; Sakka, S.; Davies, J. H.; Randell, T.; Arya, V.; Brain, C.; Tighe, M.; Allgrove, J.; Arundel, P.; Pryce, R.; Hogler, W.; Shaw, N. J. 分享 收藏
Best practice management guidelines for fibrous dysplasia/McCune-Albright syndrome: a consensus statement from the FD/MAS international consortium 纤维异常增生/mccune-albright综合征的最佳实践管理指南: FD/MAS国际联盟的共识声明 Javaid, Muhammad Kassim; Boyce, Alison; Appelman-Dijkstra, Natasha; Ong, Juling; Defabianis, Patrizia; Offiah, Amaka; Arunde, Paul; Shaw, Nick; Dal Pos, Valter; Underhil, Ann; Portero, Deanna; Heral, Lisa; Heegaard, Anne-Marie; Masi, Laura; Monsell, Fergal; Stanton, Robert; Dijkstra, Pieter Durk Sander; Brandi, Maria Luisa; Chapurlat, Roland; Hamdy, Neveen Agnes Therese; Collins, Michael Terrence 分享 收藏
Monitoring guidance for patients with hypophosphatasia treated with asfotase alfa Kishnani, Priya S.; Rush, Eric T.; Arundel, Paul; Bishop, Nick; Dahir, Kathryn; Fraser, William; Harmatz, Paul; Linglart, Agnes; Munns, Craig F.; Nunes, Mark E.; Saal, Howard M.; Seefried, Lothar; Ozono, Keiichi 分享 收藏
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Compound heterozygous variants in NBAS as a cause of atypical osteogenesis imperfecta Balasubramanian, M.; Hurst, J.; Brown, S.; Bishop, N. J.; Arundel, P.; DeVile, C.; Pollitt, R. C.; Crooks, L.; Longman, D.; Caceres, J. F.; Shacldey, F.; Connolly, S.; Payne, J. H.; Offiah, A. C.; Hughes, D.; Study, D. D. D.; Parker, M. J.; Hide, W.; Skerry, T. M. 分享 收藏
Amalgamated Reference Data for Size-Adjusted Bone Densitometry Measurements in 3598 Children and Young Adults-the ALPHABET Study Crabtree, Nicola J.; Shaw, Nicholas J.; Bishop, Nicholas J.; Adams, Judith E.; Mughal, M. Zulf; Arundel, Paul; Fewtrell, Mary S.; Ahmed, S. Faisal; Treadgold, Laura A.; Hoegler, Wolfgang; Bebbington, Natalie A.; Ward, Kate A. 分享 收藏
Risedronate in children with osteogenesis imperfecta: a randomised, double-blind, placebo-controlled trial Bishop, Nick; Adami, Silvano; Ahmed, S. Faisal; Anton, Jordi; Arundel, Paul; Burren, Christine P.; Devogelaer, Jean-Pierre; Hangartner, Thomas; Eva Hosszu; Lane, Joseph M.; Lorenc, Roman; Makitie, Outi; Munns, Craig F.; Paredes, Ana; Pavlov, Helene; Plotkin, Horacio; Raggio, Cathleen L.; Loreto Reyes, Maria; Schoenau, Eckhard; Semler, Oliver; Sillence, David O.; Steiner, Robert D. 分享 收藏
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