$RAREUltragenyx Pharmaceutical Inc.

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News

• FDA Approves First Therapy for Patients aged 8 years and older with Glycogen Storage Disease Type Ia
  The U.S. Food and Drug Administration today issued an accelerated approval for Genglycos (pariglasgene brecaparvovec-opnr) as the first-ever treatment for adults and children 8 years of age and old…
  fda_press · 3h ago
• FDA approves Regeneron’s garetosmab for disease that turns tissue to bone
  Regeneron has secured approval for its treatment for a debilitating disease that replaces muscle and connective tissue with bone. The FDA on Wednesday cleared the drugmaker’s activin A-targeting an…
  endpoints · 6h ago
• STAT+: Regeneron drug for disease that causes dangerous bone growth earns FDA approval
  A Regeneron drug that treats an ultra-rare disease that causes bone to form where it's not meant to be has won approval.
  stat · 7h ago
• Kolon TissueGene cuts 37 US staffers after Phase 3 fail
  Follow along as BioSpace tracks job cuts and restructuring initiatives.
  biospace · 11h ago
• Trump picks White House aide Heidi Overton as FDA chief: Reports
  If Heidi Overton clears the Senate nomination process, analysts say she will have a tough job ahead to “restore predictability” to an agency battered by layoffs and controversial policies.
  biospace · 12h ago
• STAT+: In wake of boy’s death, gene editing experts dissect Huidagene’s decisions
  Experts question technical improvements Huidagene tried when using a Duchenne muscular dystrophy treatment strategy largely abandoned by U.S. companies.
  stat · 16h ago
• Takeda leads wave of orexin drugs with potential to upend narcolepsy market
  The FDA’s August approval of Takeda’s Orzeyful for treating the full set of narcolepsy symptoms initiated a new era in the sleep market.
  biospace · 20h ago
• BioMarin acquires experimental drug for rare bone disorder in $275M deal
  The Alesta buyout helps restock BioMarin's pipeline with an asset it says could “reach our largest addressable patient population,” joining other muscle and skeletal disease treatments in development.
  biopharma_dive · 1d ago
• BioMarin does another small deal to get early-stage bone disease asset
  A week after ending development of a rare disease drug from a $270 million acquisition, BioMarin is back at the dealmaking table for a very similar number. The California biopharma
  endpoints · 1d ago
• Leo Pharma buys rare skin disease drug in $435M deal
  Tanabe Pharma has already asked the FDA to approve the medicine for two conditions that cause severe reactions to sunlight.
  biopharma_dive · 1d ago