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Review

Clinical Evidence Supporting Early Treatment Of Patients With Spinal Muscular Atrophy: Current Perspectives

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Pages 1153-1161 | Published online: 02 Oct 2019

Keep up to date with the latest research on this topic with citation updates for this article.

Read on this site (9)

Alzira Alves De Siqueira Carvalho, Cyril Tychon & Laurent Servais. (2023) Newborn screening for spinal muscular atrophy - what have we learned?. Expert Review of Neurotherapeutics 23:11, pages 1005-1012.
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Helgi Thor Hjartarson, Kristofer Nathorst-Böös & Thomas Sejersen. (2022) Disease Modifying Therapies for the Management of Children with Spinal Muscular Atrophy (5q SMA): An Update on the Emerging Evidence. Drug Design, Development and Therapy 16, pages 1865-1883.
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Hugh J. McMillan, Crystal M. Proud, Michelle A. Farrar, Ian E. Alexander, Francesco Muntoni & Laurent Servais. (2022) Onasemnogene abeparvovec for the treatment of spinal muscular atrophy. Expert Opinion on Biological Therapy 22:9, pages 1075-1090.
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Theodora Markati, Gemma Fisher, Sithara Ramdas & Laurent Servais. (2022) Risdiplam: an investigational survival motor neuron 2 (SMN2) splicing modifier for spinal muscular atrophy (SMA). Expert Opinion on Investigational Drugs 31:5, pages 451-461.
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Theodora Markati, Jessica Duis & Laurent Servais. (2021) Therapies in preclinical and clinical development for Angelman syndrome. Expert Opinion on Investigational Drugs 30:7, pages 709-720.
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Laurent Servais, Giovanni Baranello, Mariacristina Scoto, Aurore Daron & Maryam Oskoui. (2021) Therapeutic interventions for spinal muscular atrophy: preclinical and early clinical development opportunities. Expert Opinion on Investigational Drugs 30:5, pages 519-527.
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Maria Jędrzejowska. (2020) Advances in Newborn Screening and Presymptomatic Diagnosis of Spinal Muscular Atrophy. Degenerative Neurological and Neuromuscular Disease 10, pages 39-47.
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Maria Stefanova Kamusheva & Maria Jordanova Dimitrova. (2020) Clinical and economic assessment of nusinersen: the Bulgarian perspective. Expert Opinion on Orphan Drugs 8:10, pages 403-415.
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Sithara Ramdas & Laurent Servais. (2020) New treatments in spinal muscular atrophy: an overview of currently available data. Expert Opinion on Pharmacotherapy 21:3, pages 307-315.
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