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Ultragenyx Pharmaceutical Inc.

http://www.ultragenyx.com/

Latest From Ultragenyx Pharmaceutical Inc.

Medicare Coverage Restrictions For Expensive Drugs: Did Aduhelm Let The Genie Out Of The Bottle?

Payer discomfort with accelerated approvals exploded into the public arena with the Medicare national coverage determination on Aduhelm and similar drugs, and developers worry that the fallout will be far reaching.

Medicare Reimbursement

Medicare Coverage Restrictions For Expensive Drugs: Did Aduhelm Let The Genie Out Of The Bottle?

Payer discomfort with accelerated approvals exploded into the public arena with the Medicare national coverage determination on Aduhelm and similar drugs, and developers worry that the fallout will be far reaching.

Medicare Reimbursement

Gene Therapy Biotechs Treating Sanfilippo Syndrome Hit Cash Crunch

Despite promising clinical data and solid regulatory plans, biotech firms hoping to address the unmet need in Sanfilippo Syndrome through gene therapy are facing financial struggles.

Companies Gene Therapy

Orphalan Flies Solo In US After Wilson’s Disease Drug Win

Naseem Amin, the privately owned group’s CEO, tells Scrip that, just as in Europe, Orphalan has the resources to commercialize Cuvrior in the US: “We understand the market and we don't need external partners."

Approvals Rare Diseases
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Company Information

  • Industry
  • Biotechnology
  • Pharmaceuticals
  • Biotechnology
    • Gene Therapy, Cell Therapy
    • Large Molecule
      • Antibodies
  • Other Names / Subsidiaries
    • Ultragenyx
    • Dimension Therapeutics
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