Spinal Muscular Atrophy (SMA) Stem Cell Medical Reports
Summaries of published research on stem cell therapy for spinal muscular atrophy (sma), each linking to the original study.
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Stem Cell Research & Therapy · 2024-04-01
Administration of Adipose-Derived Stem Cells Extracellular Vesicles in a Murine Model of Spinal Muscular Atrophy: Effects of a New Potential Therapeutic Strategy
Federica Virla, Ermanna Turano, Ilaria Scambi, Lorenzo Schiaffino, Marina Boido, and Raffaella Mariotti.
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eClinicalMedicine · 2026-04-13
Safety and Efficacy of Intravenous Onasemnogene Abeparvovec Gene Therapy in Patients With Spinal Muscular Atrophy Type 1: Interim Analysis From LT-001
Megan A. Waldrop, Roberto Bernardo Escudero, Lina Yang, Rebekah A. Camarata, Emily C. Branic, Lesa Mehl, Andreja Ilić, and Anne M. Connolly.
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The Lancet Regional Health - Europe · 2024-02-06
Long-Term Efficacy and Safety of Nusinersen in Adults With 5q Spinal Muscular Atrophy: A Prospective European Multinational Observational Study
René Günther, Claudia Diana Wurster, Svenja Brakemeier, Alma Osmanovic, Olivia Schreiber-Katz, Susanne Petri, Zeljko Uzelac, Miriam Hiebeler, Simone Thiele, Maggie C. Walter, Markus Weiler, Tobias Kessler, Maren Freigang, Hanna Sophie Lapp, Isabell Cordts, Paul Lingor, Marcus Deschauer, Andreas Hahn, Kyriakos Martakis, Robert Steinbach, Benjamin Ilse, Annekathrin Rödiger, Julia Bellut, Julia Nentwich, Daniel Zeller, Mohamad Tareq Muhandes, Tobias Baum, Jan Christoph Koch, Bertold Schrank, Sophie Fischer, Andreas Hermann, Christoph Kamm, Steffen Naegel, Alexander Mensch, Markus Weber, Christoph Neuwirth, Helmar C. Lehmann, Gilbert Wunderlich, Christian Stadler, Maike Tomforde, Annette George, Martin Groß, Astrid Pechmann, Janbernd Kirschner, Matthias Türk, Mareike Schimmel, Günther Bernert, Pascal Martin, Christian Rauscher, Gerd Meyer zu Hörste, Petra Baum, Wolfgang Löscher, Marina Flotats-Bastardas, Cornelia Köhler, Kristina Probst-Schendzielorz, Susanne Goldbach, Ulrike Schara-Schmidt, Wolfgang Müller-Felber, Hanns Lochmüller, Otgonzul von Velsen, Christoph Kleinschnitz, Albert C. Ludolph, and Tim Hagenacker.
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Frontiers in Molecular Medicine · 2026-08-25
Spinal Muscular Atrophy in the Disease-Modifying Therapy Era: Successes, Limitations and Future Directions
Madison M. Sexton, Emma W. Crow, and Congyue Annie Peng.
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Neuroprotection · 2025-12-22
Therapeutic Potential of Stem Cells in Pediatric Neurology: Insights From Clinical Trials
Daniel Bou Najm and Saada Alame.
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2012-05-05
Treatment of Spinal Muscolar Atrophy with Intrathecal Mesenchymal Cells
Marino Andolina
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2011-07-21
Stem cell model of spinal muscular atrophy
Allison D. Ebert, Clive N. Svendsen
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2023-02-01
Safety and efficacy of gene therapy with onasemnogene abeparvovec in the treatment of spinal muscular atrophy
Dongling Yang, Yiyan Ruan, Yuyi Chen
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2022-01-26
Restoring SMN Expression
Tejal Aslesh, Toshifumi Yokota
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2017-10-01
Advances in modeling and treating spinal muscular atrophy
Meaghan Van Alstyne, Livio Pellizzoni
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2016-11-01
Spinal Muscular Atrophy — Kolb & Kissel
Stephen J. Kolb, M.D, John T. Kissel, M.D.
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