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Nature Methods|August 26, 2014
Adenoviral vector DNA for accurate genome editing with engineered nucleasesMaarten Holkers, Ignazio Maggio, Sara F D Henriques, et al.
Peerj|December 7, 2020
HIV-1 promoter is gradually silenced when integrated into BACH2 in Jurkat T-cellsAnne Inderbitzin, Yik Lim Kok, Lisa Jörimann, et al.
Acta Myologica : Myopathies and Cardiomyopathies : Official Journal of the Mediterranean Society of Myology|July 2, 2020
Coagulation disorders in Duchenne muscular dystrophy? Results of a registry-based online surveyDavid C Schorling, Cornelia K Müller, Astrid Pechmann, et al.
Neuromuscular Disorders : NMD|March 6, 2015
Two novel nebulin variants in an adult patient with congenital nemaline myopathyAnne K Güttsches, Gabriele Dekomien, Kristl G Claeys, et al.
Neuromuscular Disorders : NMD|November 20, 2020
Tasks and interfaces in primary and specialized palliative care for Duchenne muscular dystrophy - A patients' perspectiveMaria Janisch, Kristin Boehme, Simone Thiele, et al.
Orphanet Journal of Rare Diseases|August 13, 2025
Epidemiology, disease burden and costs of Duchenne muscular dystrophy in Germany: an observational, retrospective health claims data analysisJoanna Diesing, Janbernd Kirschner, Astrid Pechmann, et al.
Journal of Neuromuscular Diseases|September 24, 2023
A Mixed-method Approach to Develop an Ambulatory Module of the SMA Independence ScaleHannah Staunton, Sophie Cleanthous, Vanda Teodoro, et al.
Journal of Neurology|June 22, 2017
A multi-source approach to determine SMA incidence and research ready populationIngrid E C Verhaart, Agata Robertson, Rebecca Leary, et al.
Journal of Neurology|December 25, 2014
Adult care for Duchenne muscular dystrophy in the UKSunil Rodger, Katherine L Woods, Catherine L Bladen, et al.
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