日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Preventing Cardiomyopathy in Duchenne Muscular Dystrophy: Long-Term Follow-Up of Patients in the Randomised, Placebo-Controlled Drug-Trial of Perindopril and Bisoprolol

预防杜氏肌营养不良症患者发生心肌病:培哚普利和比索洛尔随机、安慰剂对照药物试验患者的长期随访

Bourke, John P; Bryant, Andrew; Landon, Gregory; Burn, Alexis; Spinty, Stefan; Quinlivan, Ros; Alhaswani, Zoya; Chadwick, Thomas; Muntoni, Francesco; Guglieri, Michela

Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial

Vamorolone治疗杜氏肌营养不良症男孩48周的疗效和安全性:一项随机对照试验

Dang, Utkarsh J; Damsker, Jesse M; Guglieri, Michela; Clemens, Paula R; Perlman, Seth J; Smith, Edward C; Horrocks, Iain; Finkel, Richard S; Mah, Jean K; Deconinck, Nicolas; Goemans, Nathalie M; Haberlová, Jana; Straub, Volker; Mengle-Gaw, Laurel; Schwartz, Benjamin D; Harper, Amy; Shieh, Perry B; De Waele, Liesbeth; Castro, Diana; Yang, Michele L; Ryan, Monique M; McDonald, Craig M; Tulinius, Mar; Webster, Richard I; Mcmillan, Hugh J; Kuntz, Nancy; Rao, Vamshi K; Baranello, Giovanni; Spinty, Stefan; Childs, Anne-Marie; Sbrocchi, Annie M; Selby, Kathryn A; Monduy, Migvis; Nevo, Yoram; Vilchez, Juan J; Nascimento-Osorio, Andres; Niks, Erik H; De Groot, Imelda J M; Katsalouli, Marina; Van Den Anker, John N; Ward, Leanne M; Leinonen, Mika; D'Alessandro, Andrea L; Hoffman, Eric P

Risdiplam in Spinal Muscular Atrophy: Safety Profile and Use Through The Early Access to Medicine Scheme for the Paediatric Cohort in Great Britain

利司他普兰治疗脊髓性肌萎缩症:安全性概况及在英国儿科人群早期药物获取计划中的应用

Cornell, Nikki; Childs, Anne-Marie; Wraige, Elizabeth; Munot, Pinki; Ambegaonkar, Gautam; Chow, Gabriel; Hughes, Imelda; Illingworth, Marjorie; Majumdar, Anirban; Marini-Bettolo, Chiara; Parasuraman, Deepak; Spinty, Stefan; Willis, Tracey; Scoto, Mariacristina; Baranello, Giovanni

Multidisciplinary Care for Moebius Syndrome and Related Disorders: Building a Management Protocol

莫比乌斯综合征及相关疾病的多学科诊疗:构建管理方案

Odedra, Amar; Blumenow, Wendy; Dainty, Jennifer; Dasgupta, Soumit; Dominguez-Gonzalez, Susana; Gonzalez-Martin, Jose; Hartley, Helen; Kelly, Maria; McKay, Victoria H; Sharma, Ravi; Spinty, Stefan; Fattah, Adel Y

Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial

不同皮质类固醇剂量方案对杜氏肌营养不良症男孩临床疗效的影响:一项随机临床试验

Guglieri, Michela; Bushby, Kate; McDermott, Michael P; Hart, Kimberly A; Tawil, Rabi; Martens, William B; Herr, Barbara E; McColl, Elaine; Speed, Chris; Wilkinson, Jennifer; Kirschner, Janbernd; King, Wendy M; Eagle, Michelle; Brown, Mary W; Willis, Tracey; Griggs, Robert C; Straub, Volker; van Ruiten, Henriette; Childs, Anne-Marie; Ciafaloni, Emma; Shieh, Perry B; Spinty, Stefan; Maggi, Lorenzo; Baranello, Giovanni; Butterfield, Russell J; Horrocks, I A; Roper, Helen; Alhaswani, Zoya; Flanigan, Kevin M; Kuntz, Nancy L; Manzur, Adnan; Darras, Basil T; Kang, Peter B; Morrison, Leslie; Krzesniak-Swinarska, Monika; Mah, Jean K; Mongini, Tiziana E; Ricci, Federica; von der Hagen, Maja; Finkel, Richard S; O'Reardon, Kathleen; Wicklund, Matthew; Kumar, Ashutosh; McDonald, Craig M; Han, Jay J; Joyce, Nanette; Henricson, Erik K; Schara-Schmidt, Ulrike; Gangfuss, Andrea; Wilichowski, Ekkehard; Barohn, Richard J; Statland, Jeffrey M; Campbell, Craig; Vita, Giuseppe; Vita, Gian Luca; Howard, James F Jr; Hughes, Imelda; McMillan, Hugh J; Pegoraro, Elena; Bello, Luca; Burnette, W Bryan; Thangarajh, Mathula; Chang, Taeun

Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial

Vamorolone 与安慰剂和泼尼松治疗杜氏肌营养不良症男孩的疗效和安全性:一项随机临床试验

Guglieri, Michela; Clemens, Paula R; Perlman, Seth J; Smith, Edward C; Horrocks, Iain; Finkel, Richard S; Mah, Jean K; Deconinck, Nicolas; Goemans, Nathalie; Haberlova, Jana; Straub, Volker; Mengle-Gaw, Laurel J; Schwartz, Benjamin D; Harper, Amy D; Shieh, Perry B; De Waele, Liesbeth; Castro, Diana; Yang, Michelle L; Ryan, Monique M; McDonald, Craig M; Tulinius, Mar; Webster, Richard; McMillan, Hugh J; Kuntz, Nancy L; Rao, Vashmi K; Baranello, Giovanni; Spinty, Stefan; Childs, Anne-Marie; Sbrocchi, Annie M; Selby, Kathryn A; Monduy, Migvis; Nevo, Yoram; Vilchez-Padilla, Juan J; Nascimento-Osorio, Andres; Niks, Erik H; de Groot, Imelda J M; Katsalouli, Marina; James, Meredith K; van den Anker, Johannes; Damsker, Jesse M; Ahmet, Alexandra; Ward, Leanne M; Jaros, Mark; Shale, Phil; Dang, Utkarsh J; Hoffman, Eric P

British Society for Rheumatology guideline on management of paediatric, adolescent and adult patients with idiopathic inflammatory myopathy

英国风湿病学会关于特发性炎症性肌病儿童、青少年和成人患者管理的指南

Oldroyd, Alexander G S; Lilleker, James B; Amin, Tania; Aragon, Octavio; Bechman, Katie; Cuthbert, Verna; Galloway, James; Gordon, Patrick; Gregory, William J; Gunawardena, Harsha; Hanna, Michael G; Isenberg, David; Jackman, John; Kiely, Patrick D W; Livermore, Polly; Machado, Pedro M; Maillard, Sue; McHugh, Neil; Murphy, Ruth; Pilkington, Clarissa; Prabu, Athiveeraramapandian; Rushe, Phoebe; Spinty, Stefan; Swan, Joanne; Tahir, Hasan; Tansley, Sarah L; Truepenny, Paul; Truepenny, Yvonne; Warrier, Kishore; Yates, Mark; Papadopoulou, Charalampia; Martin, Neil; McCann, Liza; Chinoy, Hector

Preventing Cardiomyopathy in DMD: A Randomized Placebo-Controlled Drug Trial

预防杜氏肌营养不良症(DMD)患者发生心肌病:一项随机安慰剂对照药物试验

Bourke, John P; Watson, Gillian; Spinty, Stefan; Bryant, Andrew; Roper, Helen; Chadwick, Thomas; Wood, Ruth; McColl, Elaine; Bushby, Kate; Muntoni, Francesco; Guglieri, Michela

Pathogenic variants in MT-ATP6: A United Kingdom-based mitochondrial disease cohort study

MT-ATP6 致病变异:一项基于英国的线粒体疾病队列研究

Ng, Yi Shiau; Martikainen, Mika H; Gorman, Gráinne S; Blain, Alasdair; Bugiardini, Enrico; Bunting, Apphia; Schaefer, Andrew M; Alston, Charlotte L; Blakely, Emma L; Sharma, Sunil; Hughes, Imelda; Lim, Albert; de Goede, Christian; McEntagart, Meriel; Spinty, Stefan; Horrocks, Iain; Roberts, Mark; Woodward, Cathy E; Chinnery, Patrick F; Horvath, Rita; Nesbitt, Victoria; Fratter, Carl; Poulton, Joanna; Hanna, Michael G; Pitceathly, Robert D S; Taylor, Robert W; Turnbull, Doug M; McFarland, Robert

Randomised placebo-controlled trial of combination ACE inhibitor and beta-blocker therapy to prevent cardiomyopathy in children with Duchenne muscular dystrophy? (DMD Heart Protection Study): a protocol study

一项随机安慰剂对照试验,旨在评估ACE抑制剂联合β受体阻滞剂治疗预防杜氏肌营养不良症患儿心肌病的效果(DMD心脏保护研究):一项方案研究

Bourke, John P; Watson, Gillian; Muntoni, Francesco; Spinty, Stefan; Roper, Helen; Guglieri, Michela; Speed, Chris; McColl, Elaine; Chikermane, Ashish; Jayawant, Sandeep; Adwani, Satish; Willis, Tracey; Wilkinson, Jennifer; Bryant, Andrew; Chadwick, Thomas; Wood, Ruth; Bushby, Kate