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Lakshmi Balaji, Michelle A Farrar, Arlene M D’Silva & Didu S Kariyawasam. (2023) Decision-making and challenges within the evolving treatment algorithm in spinal muscular atrophy: a clinical perspective. Expert Review of Neurotherapeutics 23:7, pages 571-586.
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André Motta-Santos, Kenya Noronha, Carla Reis, Daniela Freitas, Lélia Carvalho & Mônica Andrade. (2024) Cost-Effectiveness of Technologies for the Treatment of Spinal Muscular Atrophy: A Systematic Review of Economic Studies. Value in Health Regional Issues 42, pages 100985.
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Brígida Dias Fernandes, Bárbara Corrêa Krug, Fernanda D’Athayde Rodrigues, Hérica Núbia Cardoso Cirilo, Stéfani Sousa Borges, Ida Vanessa D. Schwartz, Livia Fernandes Probst & Ivan Zimmermann. (2024) Efficacy and safety of onasemnogene abeparvovec for the treatment of patients with spinal muscular atrophy type 1: A systematic review with meta-analysis. PLOS ONE 19:5, pages e0302860.
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Brígida Dias Fernandes, Fernanda D’Athayde Rodrigues, Hérica Núbia Cardoso Cirilo, Stéfani Sousa Borges, Bárbara Corrêa Krug, Livia Fernandes Probst & Ivan Zimmermann. (2024) Cost-Effectiveness of Onasemnogene Abeparvovec Compared With Nusinersen and Risdiplam in Patients With Spinal Muscular Atrophy Type 1 in Brazil. Value in Health Regional Issues 40, pages 108-117.
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Qing Xie, Xiupeng Chen, Hong Ma, Yunxiang Zhu, Yijie Ma, Leila Jalinous, Gerald F Cox, Fiona Weaver, Jun Yang, Zachary Kennedy, Alisha Gruntman, Ailing Du, Qin Su, Ran He, Phillip WL Tai, Guangping Gao & Jun Xie. (2024) Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor. EMBO Molecular Medicine 16:4, pages 945-965.
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Victoria W. Dayer, Michael F. Drummond, Omar Dabbous, Mondher Toumi, Peter Neumann, Sean Tunis, Nelson Teich, Shadi Saleh, Ulf Persson, Johann-Matthias Graf von der Schulenburg, Daniel C. Malone, Tay Salimullah & Sean D. Sullivan. (2024) Real-world evidence for coverage determination of treatments for rare diseases. Orphanet Journal of Rare Diseases 19:1.
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Itay Tokatly Latzer, Liora Sagi, Revital Lavi, Sharon Aharoni, Jacob Bistritzer, Iris Noyman, Mira Ginsburg, Angela Lev-Or, Sharona Katzenellenbogen, Yoram Nevo & Aviva Fattal-Valevski. (2023) Real-Life Outcome After Gene Replacement Therapy for Spinal Muscular Atrophy: A Multicenter Experience. Pediatric Neurology 144, pages 60-68.
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Tammy Jiang, Bora Youn, Angela D. Paradis, Rachel Beckerman, Lianne Barnieh & Nicole B. Johnson. (2023) A Critical Appraisal of Matching-Adjusted Indirect Comparisons in Spinal Muscular Atrophy. Advances in Therapy 40:7, pages 2985-3005.
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Carmen Leon-Astudillo, Barry J. Byrne & Ramzi G. Salloum. (2022) Addressing the implementation gap in advanced therapeutics for spinal muscular atrophy in the era of newborn screening programs. Frontiers in Neurology 13.
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Hannah A. Blair. (2022) Onasemnogene Abeparvovec: A Review in Spinal Muscular Atrophy. CNS Drugs 36:9, pages 995-1005.
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Min Yang, Hiroyuki Awano, Satoru Tanaka, Walter Toro, Su Zhang, Omar Dabbous & Ataru Igarashi. (2022) Systematic Literature Review of Clinical and Economic Evidence for Spinal Muscular Atrophy. Advances in Therapy 39:5, pages 1915-1958.
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Ming-Jen Lee, Inyoul Lee & Kai Wang. (2022) Recent Advances in RNA Therapy and Its Carriers to Treat the Single-Gene Neurological Disorders. Biomedicines 10:1, pages 158.
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