Rare Disease

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nothing on the calendar yet

News(10)

• 7 biopharma M&A deals where layoffs followed
  Workforce cuts this year at seven companies involved in recent mergers and acquisitions will cost over 1,300 employees their jobs. Affected businesses include Arcellx and Tourmaline Bio, acquired b…
  biospace · 1h ago
• Why pediatric rare oncology outperforms in a volatile biotech market
  Real progress happens where unmet medical need is greatest and patient impact can be clearly measured. Pediatric rare cancers exemplify these criteria and have therefore generated continued investm…
  biospace · 4h 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 · 15h ago
• Kolon TissueGene cuts 37 US staffers after Phase 3 fail
  Follow along as BioSpace tracks job cuts and restructuring initiatives.
  biospace · 18h 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 · 20h 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 · 1d 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 adds to dermatology portfolio with $435M deal for late-stage rare disease drug
  This is the third recent acquisition conducted by Denmark’s LEO Pharma in medical dermatology, a portfolio that marked 12% growth in the first half of the year.
  biospace · 1d ago
• Amylyx eyes FDA filing for GLP-1 blocker after late-stage win in post-bariatric hypoglycemia
  With a Phase 3 trial showing a reduction of serious and severe hypoglycemic episodes in patients who have undergone weight loss surgery, Amylyx Pharmaceuticals plans to file for approval this year.…
  biospace · 1d ago