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Compilation of online resources of relevance to 'Spinraza and advanced therapies:a stakeholder special' issue of Gene Therapy [PDF]
, 2017 Prakash, V, Yáñez-Muñoz, R J
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Why antisense could make sense for neurodegeneration [PDF]
, 2017 Massey, Thomas, Robertson, Neil
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Protein production is an early biomarker for RNA‐targeted therapies [PDF]
, 2018 Alex, Jacob +10 more
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Therapeutic Role of Nusinersen on Respiratory Progression in Pediatric Patients With Spinal Muscular Atrophy Type 2 and Nonambulant Type 3 [PDF]
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NCALD Antisense Oligonucleotide Therapy in Addition to Nusinersen further Ameliorates Spinal Muscular Atrophy in Mice [PDF]
, 2019 Laura Torres‐Benito +9 more
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Gene Therapy, more than ever-a new vision for the journal [PDF]
, 2017 Yáñez-Muñoz, R J
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Radiation dose reduction for CT-guided intrathecal nusinersen administration in adult patients with spinal muscular atrophy [PDF]
, 2020 Isabell Cordts +7 more
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