tofersen sodium

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News(4)

• Ionis-Otsuka offer 'groundbreaking' improvement in function and survival for genetic type of ALS
  Ulefnersen, an RNA-targeted therapy being developed by Ionis Pharmaceuticals and Otsuka Pharmaceutical, improved measurements of functional impairment and survival in patients with a rare, genetic…
  biospace · 13d ago
• Ionis’ ‘breakthrough’ Alexander disease approval establishes pillar of rare disease pipeline
  After Jenny Pearson’s daughter Elise first had a seizure at 16 months old, an MRI came back clean—her brain “looked beautiful,” Pearson recalled to Fierce. But a follow-up scan a year later, just d…
  fiercebiotech · 1mo ago
• FDA approves Ionis’ antisense drug as first targeted therapy for Alexander disease
  Zanvastro, an antisense oligonucleotide therapy, represents the first independent neurology launch for Ionis Pharmaceuticals, which intends to leverage the foothold for more approvals in rare neuro…
  biospace · 1mo ago
• Biogen’s targeted ALS treatment is reversing decline in some patients. Can more be helped?
  Three years after Qalsody was approved by the FDA as the first drug to target a genetic cause of ALS, some patients are reporting not only disease stabilization but also improvement of symptoms.
  biospace · 1mo ago