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Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial.
Dang UJ, Damsker JM, Guglieri M, Clemens PR, Perlman SJ, Smith EC, Horrocks I, Finkel RS, Mah JK, Deconinck N, Goemans NM, Haberlová J, Straub V, Mengle-Gaw L, Schwartz BD, Harper A, Shieh PB, De Waele L, Castro D, Yang ML, Ryan MM, McDonald CM, Tulinius M, Webster RI, Mcmillan HJ, Kuntz N, Rao VK, Baranello G, Spinty S, Childs AM, Sbrocchi AM, Selby KA, Monduy M, Nevo Y, Vilchez JJ, Nascimento-Osorio A, Niks EH, De Groot IJM, Katsalouli M, Van Den Anker JN, Ward LM, Leinonen M, D'Alessandro AL, Hoffman EP. Dang UJ, et al. Among authors: childs am. Neurology. 2024 Mar 12;102(5):e208112. doi: 10.1212/WNL.0000000000208112. Epub 2024 Feb 9. Neurology. 2024. PMID: 38335499 Free PMC article. Clinical Trial.
Risdiplam in Spinal Muscular Atrophy: Safety Profile and Use Through The Early Access to Medicine Scheme for the Paediatric Cohort in Great Britain.
Cornell N, Childs AM, Wraige E, Munot P, Ambegaonkar G, Chow G, Hughes I, Illingworth M, Majumdar A, Marini-Bettolo C, Parasuraman D, Spinty S, Willis T, Scoto M, Baranello G; Paediatric UK Risdiplam EAMS Working Group. Cornell N, et al. Among authors: childs am. J Neuromuscul Dis. 2024;11(2):361-368. doi: 10.3233/JND-230162. J Neuromuscul Dis. 2024. PMID: 38189761 Free PMC article.
Development of respiratory care guidelines for Duchenne muscular dystrophy in the UK: key recommendations for clinical practice.
Childs AM, Turner C, Astin R, Bianchi S, Bourke J, Cunningham V, Edel L, Edwards C, Farrant P, Heraghty J, James M, Massey C, Messer B, Michel Sodhi J, Murphy PB, Schiava M, Thomas A, Trucco F, Guglieri M. Childs AM, et al. Thorax. 2024 Apr 15;79(5):476-485. doi: 10.1136/thorax-2023-220811. Thorax. 2024. PMID: 38123347 Free PMC article. Review.
Reporting of paediatric osteoporotic vertebral fractures in Duchenne muscular dystrophy and potential impact on clinical management: the need for standardised and structured reporting.
Martin H, Henderson A, Allen R, Childs AM, Dunne J, Horrocks I, Joseph S, Kraft JK, Ward K, Mushtaq T, Mason A, Kyriakou A, Wong SC. Martin H, et al. Among authors: childs am. Pediatr Radiol. 2024 Jan;54(1):117-126. doi: 10.1007/s00247-023-05805-4. Epub 2023 Dec 11. Pediatr Radiol. 2024. PMID: 38072887 Free PMC article.
Characteristics and outcomes of neonates hospitalised with SARS-CoV-2 infection in the UK by variant: a prospective national cohort study.
Gale C, Sharkey D, Fitzpatrick KE, Mactier H, Morelli A, Nakahara M, Hurd M, Placzek A, Knight M, Ladhani SN, Draper ES, Doherty C, Quigley MA, Kurinczuk JJ; Neonatal complications of COVID-19 Collaborative Group. Gale C, et al. Arch Dis Child Fetal Neonatal Ed. 2024 Apr 18;109(3):279-286. doi: 10.1136/archdischild-2023-326167. Arch Dis Child Fetal Neonatal Ed. 2024. PMID: 37968087 Free PMC article.
274 results