Nusinersen

Nusinersen
Product Description

Nusinersen is a modified antisense oligonucleotide (ASO) administered intrathecally. It binds to the ISS-N1 sequence in intron 7 of SMN2 pre-mRNA, promoting exon 7 inclusion and production of full-length functional SMN protein. Approved across all SMA phenotypes including Type I, II, III, and presymptomatic individuals, Nusinersen has demonstrated significant improvements in motor function and survival, fundamentally altering the natural history of this previously untreatable neuromuscular disorder.

QILU PHARMACEUTICAL CO., LTD.

  • Country/Region:
    China
  • On CPHI since:
    2015
  • Employees:
    5000+
Company types
Manufacturer/Innovator
Specifications
  • Selling Points
    Small Nucleic Acid

QILU PHARMACEUTICAL CO., LTD.

  • Country/Region:
    China
  • On CPHI since:
    2015
  • Employees:
    5000+
Company types
Manufacturer/Innovator

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