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Year Number of Results
1966 1
1967 1
1986 3
1987 2
1988 6
1989 1
1990 3
1996 1
1999 1
2001 1
2002 1
2003 1
2008 1
2009 2
2010 3
2011 1
2012 3
2013 4
2014 4
2015 5
2016 3
2017 5
2018 8
2019 7
2020 3
2021 5
2022 9
2023 7
2024 3

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83 results

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Page 1
Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management.
Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, Kaul A, Kinnett K, McDonald C, Pandya S, Poysky J, Shapiro F, Tomezsko J, Constantin C; DMD Care Considerations Working Group. Bushby K, et al. Lancet Neurol. 2010 Jan;9(1):77-93. doi: 10.1016/S1474-4422(09)70271-6. Epub 2009 Nov 27. Lancet Neurol. 2010. PMID: 19945913 Review.
Translational and regulatory challenges for exon skipping therapies.
Aartsma-Rus A, Ferlini A, Goemans N, Pasmooij AM, Wells DJ, Bushby K, Vroom E, Balabanov P. Aartsma-Rus A, et al. Among authors: vroom e. Hum Gene Ther. 2014 Oct;25(10):885-92. doi: 10.1089/hum.2014.086. Hum Gene Ther. 2014. PMID: 25184444 Review.
EURO-NMD registry: federated FAIR infrastructure, innovative technologies and concepts of a patient-centred registry for rare neuromuscular disorders.
Atalaia A, Wandrei D, Lalout N, Thompson R, Tassoni A, 't Hoen PAC, Athanasiou D, Baker SA, Sakellariou P, Paliouras G, D'Angelo C, Horvath R, Mancuso M, van der Beek N, Kornblum C, Kirschner J, Pareyson D, Bassez G, Blacas L, Jacoupy M, Eng C, Lamy F, Plançon JP, Haberlova J, Brusse E, Hoeijmakers JGJ, de Visser M, Claeys KG, Paradas C, Toscano A, Silani V, Gyenge M, Reviers E, Hamroun D, Vroom E, Wilkinson MD, Lochmuller H, Evangelista T. Atalaia A, et al. Among authors: vroom e. Orphanet J Rare Dis. 2024 Feb 14;19(1):66. doi: 10.1186/s13023-024-03059-3. Orphanet J Rare Dis. 2024. PMID: 38355534 Free PMC article. Review.
Patient reported outcome measure for upper limb in Duchenne muscular dystrophy: correlation with PUL2.0.
Cicala G, Pane M, Coratti G, Brogna C, Fanelli L, Norcia G, Forcina N, Mazzone E, Stanca G, Ferrante R, Vento A, Ferraroli E, Ricci M, Capasso A, Leone D, Palermo C, Berti B, Cutrona C, Mahyew A, Duong T, Goemans N, Vroom E, Mercuri E. Cicala G, et al. Among authors: vroom e. Neuromuscul Disord. 2023 Sep;33(9):69-73. doi: 10.1016/j.nmd.2023.07.003. Epub 2023 Jul 22. Neuromuscul Disord. 2023. PMID: 37612177 Free article.
The Role of Patient Involvement When Developing Therapies.
Aartsma-Rus A, Vroom E, O'Reilly D. Aartsma-Rus A, et al. Among authors: vroom e. Nucleic Acid Ther. 2022 Apr;32(2):118-122. doi: 10.1089/nat.2021.0048. Epub 2021 Oct 1. Nucleic Acid Ther. 2022. PMID: 34597188 Free PMC article.
263rd ENMC International Workshop: Focus on female carriers of dystrophinopathy: refining recommendations for prevention, diagnosis, surveillance, and treatment. Hoofddorp, The Netherlands, 13-15 May 2022.
Sarkozy A, Quinlivan R, Bourke JP, Ferlini A; ENMC 263rd Workshop Study Group. Sarkozy A, et al. Neuromuscul Disord. 2023 Mar;33(3):274-284. doi: 10.1016/j.nmd.2023.01.003. Epub 2023 Jan 12. Neuromuscul Disord. 2023. PMID: 36804616 No abstract available.
Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy.
Straub V, Balabanov P, Bushby K, Ensini M, Goemans N, De Luca A, Pereda A, Hemmings R, Campion G, Kaye E, Arechavala-Gomeza V, Goyenvalle A, Niks E, Veldhuizen O, Furlong P, Stoyanova-Beninska V, Wood MJ, Johnson A, Mercuri E, Muntoni F, Sepodes B, Haas M, Vroom E, Aartsma-Rus A. Straub V, et al. Among authors: vroom e. Lancet Neurol. 2016 Jul;15(8):882-890. doi: 10.1016/S1474-4422(16)30035-7. Lancet Neurol. 2016. PMID: 27302365 Review.
Development of the Performance of the Upper Limb module for Duchenne muscular dystrophy.
Mayhew A, Mazzone ES, Eagle M, Duong T, Ash M, Decostre V, Vandenhauwe M, Klingels K, Florence J, Main M, Bianco F, Henrikson E, Servais L, Campion G, Vroom E, Ricotti V, Goemans N, McDonald C, Mercuri E; Performance of the Upper Limb Working Group. Mayhew A, et al. Among authors: vroom e. Dev Med Child Neurol. 2013 Nov;55(11):1038-45. doi: 10.1111/dmcn.12213. Epub 2013 Aug 1. Dev Med Child Neurol. 2013. PMID: 23902233 Free article. Review.
83 results