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Molecular Therapy : the Journal of the American Society of Gene Therapy|December 15, 2015
Streptococcus thermophilus CRISPR-Cas9 Systems Enable Specific Editing of the Human GenomeMaximilian Müller, Ciaran M Lee, Giedrius Gasiunas, et al.Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research|March 12, 2022
Development and Pilot Test of the Registry Evaluation and Quality Standards Tool: An Information Technology-Based Tool to Support and Review RegistriesAlexander Allen, Hannah Patrick, Jorg Ruof, et al.Pediatric Critical Care Medicine : a Journal of the Society of Critical Care Medicine and the World Federation of Pediatric Intensive and Critical Care Societies|November 17, 2016
Long-Term Follow-Up on Health-Related Quality of Life After Mechanical Circulatory Support in ChildrenThilo P K Fleck, Georg Dangel, Felix Bächle, et al.Journal of Neuromuscular Diseases|May 17, 2024
Increased Diagnostic Yield by Reanalysis of Whole Exome Sequencing Data in Mitochondrial DiseaseCatarina Olimpio, Ida Paramonov, Leslie Matalonga, et al.CNS Drugs|January 26, 2022
Restoration of Nusinersen Levels Following Treatment Interruption in People With Spinal Muscular Atrophy: Simulations Based on a Population Pharmacokinetic ModelDrew MacCannell, Zdenek Berger, Janbernd Kirschner, et al.Human Gene Therapy|October 11, 2011
Versatile and efficient genome editing in human cells by combining zinc-finger nucleases with adeno-associated viral vectorsEva-Maria Händel, Katharina Gellhaus, Kafaitullah Khan, et al.Nucleic Acids Research|January 1, 2013
Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cellsMaarten Holkers, Ignazio Maggio, Jin Liu, et al.Neuromuscular Disorders : NMD|June 26, 2009
Severe nemaline myopathy associated with consecutive mutations E74D and H75Y on a single ACTA1 alleleNatalia Garcia-Angarita, Janbernd Kirschner, Mandy Heiliger, et al.Genetics in Medicine : Official Journal of the American College of Medical Genetics|December 5, 2025
Economic evaluation of next-generation sequencing technologies in pediatric patient groups with confirmed or possible rare diseases: A systematic literature reviewMarianna De Stefano, Rudolf van Olden, Elnaz Arjmand, et al.Journal of Neuromuscular Diseases|August 20, 2025
Physiotherapeutic management of patients with SMA: A questionnaire-based online survey among physiotherapists within the SMArtCARE networkSibylle Vogt, Cornelia Voigt-Müller, Heidi Rochau-Trumpp, et al.Pageof 32