Rare Diseases

Rare Diseases Perspective


Rare Diseases News & Journal Articles

  • Recordati to Seek Canadian Approval for Kidney Disease Drug Italy's Recordati said on Tuesday it plans to seek Canadian approval for its drug Cystagon, which treats a rare kidney disease, in a potential blow to Horizon Pharma, which makes the only drug approved for treatment in Canada.
  • Biogen SMA Drug Price, Novartis Estimates for Its Treatment Far Too High -U.S. Group Biogen Inc should slash the price of its spinal muscular atrophy (SMA) drug, and the $4 million to $5 million Novartis has said its experimental gene therapy for the disease is worth is excessive, an independent U.S. organization that reviews the value of medical treatments said on Wednesday.

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