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Frontiers in Pediatrics|November 30, 2023
High-sensitive cardiac troponin I (hs-cTnI) concentrations in newborns diagnosed with spinal muscular atrophyJessika Johannsen, Deike Weiss, Joenna Driemeyer, et al.
Journal of Neurology|February 9, 2017
Predictors of Health-Related Quality of Life in boys with Duchenne muscular dystrophy from six European countriesChristiane Otto, Birgit F Steffensen, Ann-Lisbeth Højberg, et al.
Nucleic Acids Research|April 18, 2023
A novel Cas9 fusion protein promotes targeted genome editing with reduced mutational burden in primary human cellsAntonio Carusillo, Sibtain Haider, Raul Schäfer, et al.
European Urology Oncology|April 17, 2023
Adaptive Immunity in Genitourinary CancersMadhuri Koti, Trinity Bivalacqua, Peter C Black, et al.
European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society|August 9, 2020
European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophyJanbernd Kirschner, Nina Butoianu, Nathalie Goemans, et al.
Journal of Comparative Effectiveness Research|August 16, 2019
Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE RegistryFrancesco Muntoni, Isabelle Desguerre, Michela Guglieri, et al.
Frontiers in Genome Editing|March 27, 2023
High-efficiency editing in hematopoietic stem cells and the HUDEP-2 cell line based on in vitro mRNA synthesisNikoletta Y Papaioannou, Petros Patsali, Basma Naiisseh, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 16, 2025
Pilocytic astrocytoma in a child with spinal muscular atrophy treated with onasemnogene abeparvovecDorothea Holzwarth, Gabriele Calaminus, Johannes Friese, et al.
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