Biogen Further Expands Presence in China with Approval of SPINRAZA® (nusinersen), the First and Only Treatment for Spinal Muscular Atrophy | Biogen [online]. [Cit. 2023-08-25]. Dostupné online. Archivované 2023-08-25 z originálu. (po anglicky)
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How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy. Gene Therapy, 2019, s. 520–526. DOI: 10.1038/gt.2017.34. PMID 28485722.
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Biogen's SPINRAZA™ (nusinersen) Receives Notice of Compliance from Health Canada for the Treatment of 5q Spinal Muscular Atrophy (SMA) [online]. Cision, 2017-07-04. Dostupné online.Archivované 2017-12-04 na Wayback Machine
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ISS-N1 makes the First FDA-approved Drug for Spinal Muscular Atrophy. Translational Neuroscience, January 2017, s. 1–6. DOI: 10.1515/tnsci-2017-0001. PMID 28400976.
Molecular therapeutic strategies for spinal muscular atrophies: current and future clinical trials. Clinical Therapeutics, January 2014, s. 128–40. DOI: 10.1016/j.clinthera.2013.11.006. PMID 24360800.
Dual masking of specific negative splicing regulatory elements resulted in maximal exon 7 inclusion of SMN2 gene. Molecular Therapy, April 2014, s. 854–61. DOI: 10.1038/mt.2013.276. PMID 24317636. The sequence of nusinersen (UCACUUUCAUAAUGCUGG) is listed as N1-Hua in Table I.
Big win possible for Ionis/Biogen antisense drug in muscular atrophy. Nature Biotechnology, October 2016, s. 1002–1003. DOI: 10.1038/nbt1016-1002. PMID 27727217.
How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy. Gene Therapy, 2019, s. 520–526. DOI: 10.1038/gt.2017.34. PMID 28485722.
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CSHL FDA approval of life-saving SMA drug is hailed by its researcher-inventor at CSHL. Cold Spring Harbor Laboratory, 24 December 2016. Dostupné online [cit. 2017-01-01]. Archivované 2017-01-01 na Wayback Machine
Biogen's SPINRAZA™ (nusinersen) Receives Notice of Compliance from Health Canada for the Treatment of 5q Spinal Muscular Atrophy (SMA) [online]. Cision, 2017-07-04. Dostupné online.Archivované 2017-12-04 na Wayback Machine
Biogen Further Expands Presence in China with Approval of SPINRAZA® (nusinersen), the First and Only Treatment for Spinal Muscular Atrophy | Biogen [online]. [Cit. 2023-08-25]. Dostupné online. Archivované 2023-08-25 z originálu. (po anglicky)
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