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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: willis t. J Neuromuscul Dis. 2024;11(2):361-368. doi: 10.3233/JND-230162. J Neuromuscul Dis. 2024. PMID: 38189761 Free PMC article.
Developing standardized corticosteroid treatment for Duchenne muscular dystrophy.
Guglieri M, Bushby K, McDermott MP, Hart KA, Tawil R, Martens WB, Herr BE, McColl E, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Willis T, Hirtz D, Shieh PB, Straub V, Childs AM, Ciafaloni E, Butterfield RJ, Horrocks I, Spinty S, Flanigan KM, Kuntz NL, Baranello G, Roper H, Morrison L, Mah JK, Manzur AY, McDonald CM, Schara U, von der Hagen M, Barohn RJ, Campbell C, Darras BT, Finkel RS, Vita G, Hughes I, Mongini T, Pegoraro E, Wicklund M, Wilichowski E, Bryan Burnette W, Howard JF, McMillan HJ, Thangarajh M, Griggs RC. Guglieri M, et al. Among authors: willis t. Contemp Clin Trials. 2017 Jul;58:34-39. doi: 10.1016/j.cct.2017.04.008. Epub 2017 Apr 24. Contemp Clin Trials. 2017. PMID: 28450193 Free PMC article. Clinical Trial.
Mobility shift of beta-dystroglycan as a marker of GMPPB gene-related muscular dystrophy.
Sarkozy A, Torelli S, Mein R, Henderson M, Phadke R, Feng L, Sewry C, Ala P, Yau M, Bertoli M, Willis T, Hammans S, Manzur A, Sframeli M, Norwood F, Rakowicz W, Radunovic A, Vaidya SS, Parton M, Walker M, Marino S, Offiah C, Farrugia ME, Mamutse G, Marini-Bettolo C, Wraige E, Beeson D, Lochmüller H, Straub V, Bushby K, Barresi R, Muntoni F. Sarkozy A, et al. Among authors: willis t. J Neurol Neurosurg Psychiatry. 2018 Jul;89(7):762-768. doi: 10.1136/jnnp-2017-316956. Epub 2018 Feb 3. J Neurol Neurosurg Psychiatry. 2018. PMID: 29437916
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.
Bourke JP, Watson G, Muntoni F, Spinty S, Roper H, Guglieri M, Speed C, McColl E, Chikermane A, Jayawant S, Adwani S, Willis T, Wilkinson J, Bryant A, Chadwick T, Wood R, Bushby K; DMD Heart Protection study group. Bourke JP, et al. Among authors: willis t. BMJ Open. 2018 Dec 19;8(12):e022572. doi: 10.1136/bmjopen-2018-022572. BMJ Open. 2018. PMID: 30573480 Free PMC article.
Respiratory Trajectories in Type 2 and 3 Spinal Muscular Atrophy in the iSMAC Cohort Study.
Trucco F, Ridout D, Scoto M, Coratti G, Main ML, Muni Lofra R, Mayhew AG, Montes J, Pane M, Sansone V, Albamonte E, D'Amico A, Bertini E, Messina S, Bruno C, Parasuraman D, Childs AM, Gowda V, Willis T, Ong M, Marini-Bettolo C, De Vivo DC, Darras BT, Day J, Kichula EA, Mayer OH, Navas Nazario AA, Finkel RS, Mercuri E, Muntoni F; International SMA Consortium (iSMAc). Trucco F, et al. Among authors: willis t. Neurology. 2021 Jan 26;96(4):e587-e599. doi: 10.1212/WNL.0000000000011051. Epub 2020 Oct 16. Neurology. 2021. PMID: 33067401 Free PMC article.
Prediagnosis pathway benchmarking audit in patients with Duchenne muscular dystrophy.
Gowda VL, Fernandez M, Prasad M, Childs AM, Hughes I, Tirupathi S, De Goede CGEL, O'Rourke D, Parasuraman D, Willis T, Saberian S, Davidson I. Gowda VL, et al. Among authors: willis t. Arch Dis Child. 2022 Feb;107(2):160-165. doi: 10.1136/archdischild-2020-321451. Epub 2021 Jul 8. Arch Dis Child. 2022. PMID: 34244165 Free PMC article.
Adult North Star Network (ANSN): Consensus Guideline For The Standard Of Care Of Adults With Duchenne Muscular Dystrophy.
Quinlivan R, Messer B, Murphy P, Astin R, Mukherjee R, Khan J, Emmanuel A, Wong SC, Kulshresha R, Willis T, Pattni J, Willis D, Morgan A, Savvatis K, Keen R, Bourke J, Marini Bettolo C, Hewamadduma C; ANSN. Quinlivan R, et al. Among authors: willis t, willis d. J Neuromuscul Dis. 2021;8(6):899-926. doi: 10.3233/JND-200609. J Neuromuscul Dis. 2021. PMID: 34511509 Free PMC article.
293 results