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Page 1
Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Henzi BC, Schmidt S, Nagy S, Rubino-Nacht D, Schaedelin S, Putananickal N, Stimpson G; North Star Consortium; Amthor H, Childs AM, Deconinck N, de Groot I, Horrocks I, Houwen-van Opstal S, Laugel V, Lopez Lobato M, Madruga Garrido M, Nascimento Osorio A, Schara-Schmidt U, Spinty S, von Moers A, Lawrence F, Hafner P, Dorchies OM, Fischer D. Henzi BC, et al. Among authors: stimpson g. Lancet Neurol. 2023 Oct;22(10):890-899. doi: 10.1016/S1474-4422(23)00285-5. Lancet Neurol. 2023. PMID: 37739572 Clinical Trial.
Growth pattern trajectories in boys with Duchenne muscular dystrophy.
Stimpson G, Raquq S, Chesshyre M, Fewtrell M, Ridout D, Sarkozy A, Manzur A, Ayyar Gupta V, De Amicis R, Muntoni F, Baranello G; NorthStar Network. Stimpson G, et al. Orphanet J Rare Dis. 2022 Jan 24;17(1):20. doi: 10.1186/s13023-021-02158-9. Orphanet J Rare Dis. 2022. PMID: 35073949 Free PMC article.
Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial.
Mah JK, Clemens PR, Guglieri M, Smith EC, Finkel RS, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, McDonald CM, Damsker JM, Schwartz BD, Mengle-Gaw LJ, Jackowski S, Stimpson G, Ridout DA, Ayyar-Gupta V, Baranello G, Manzur AY, Muntoni F, Gordish-Dressman H, Leinonen M, Ward LM, Hoffman EP, Dang UJ; NorthStar UK Network and CINRG DNHS Investigators. Mah JK, et al. Among authors: stimpson g. JAMA Netw Open. 2022 Jan 4;5(1):e2144178. doi: 10.1001/jamanetworkopen.2021.44178. JAMA Netw Open. 2022. PMID: 35076703 Free PMC article. Clinical Trial.
Evolution of bulbar function in spinal muscular atrophy type 1 treated with nusinersen.
Weststrate H, Stimpson G, Thomas L, Scoto M, Johnson E, Stewart A, Muntoni F, Baranello G, Conway E; SMA p-FOIS Working Group*. Weststrate H, et al. Among authors: stimpson g. Dev Med Child Neurol. 2022 Jul;64(7):907-914. doi: 10.1111/dmcn.15171. Epub 2022 Feb 1. Dev Med Child Neurol. 2022. PMID: 35103306 Free PMC article.
2-Year Change in Revised Hammersmith Scale Scores in a Large Cohort of Untreated Paediatric Type 2 and 3 SMA Participants.
Stimpson G, Ramsey D, Wolfe A, Mayhew A, Scoto M, Baranello G, Muni Lofra R, Main M, Milev E, Coratti G, Pane M, Sansone V, D'Amico A, Bertini E, Messina S, Bruno C, Albamonte E, Mazzone ES, Montes J, Glanzman AM, Zolkipli-Cunningham Z, Pasternak A, Duong T, Dunaway Young S, Civitello M, Marini-Bettolo C, Day JW, Darras BT, De Vivo DC, Finkel RS, Mercuri E, Muntoni F; International SMA Consortium (iSMAc). Stimpson G, et al. J Clin Med. 2023 Feb 28;12(5):1920. doi: 10.3390/jcm12051920. J Clin Med. 2023. PMID: 36902710 Free PMC article.
Muscle magnetic resonance imaging involvement patterns in nemaline myopathies.
Perry L, Stimpson G, Singh L, Morrow JM, Shah S, Baranello G, Muntoni F, Sarkozy A. Perry L, et al. Among authors: stimpson g. Ann Clin Transl Neurol. 2023 Jul;10(7):1219-1229. doi: 10.1002/acn3.51816. Epub 2023 Jun 2. Ann Clin Transl Neurol. 2023. PMID: 37265148 Free PMC article.
269th ENMC international workshop: 10 years of clinical trials in Duchenne muscular dystrophy - What have we learned? 9-11 December 2022, Hoofddorp, The Netherlands.
Naarding KJ, Stimpson G, Ward SJ, Goemans N, McDonald C, Mercuri E, Muntoni F; 269th ENMC workshop participants. Naarding KJ, et al. Among authors: stimpson g. Neuromuscul Disord. 2023 Nov;33(11):897-910. doi: 10.1016/j.nmd.2023.10.003. Epub 2023 Oct 11. Neuromuscul Disord. 2023. PMID: 37926638
Quantifying Variability in Motor Function in Duchenne Muscular Dystrophy: UK Centiles for the NorthStar Ambulatory Assessment, 10 m Walk Run Velocity and Rise from Floor Velocity in GC Treated Boys.
Stimpson G, Ridout D, Wolfe A, Milev E, O'Reilly E, Manzur A, Sarkozy A, Muntoni F, Cole TJ, Baranello G; NorthStar Network. Stimpson G, et al. J Neuromuscul Dis. 2024;11(1):153-166. doi: 10.3233/JND-230159. J Neuromuscul Dis. 2024. PMID: 37980680 Free PMC article.
Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3.
Wolfe A, Stimpson G, Ramsey D, Coratti G, Dunaway Young S, Mayhew A, Pane M, Rohwer A, Muni Lofra R, Duong T, O'Reilly E, Milev E, Civitello M, Sansone VA, D'Amico A, Bertini E, Messina S, Bruno C, Albamonte E, Mazzone E, Main M, Montes J, Glanzman AM, Zolkipli-Cunningham Z, Pasternak A, Marini-Bettolo C, Day JW, Darras BT, De Vivo DC, Baranello G, Scoto M, Finkel RS, Mercuri E, Muntoni F; international SMA consortium (iSMAc). Wolfe A, et al. Among authors: stimpson g. J Neuromuscul Dis. 2024;11(3):665-677. doi: 10.3233/JND-230211. J Neuromuscul Dis. 2024. PMID: 38427497 Free PMC article.
13 results