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Welcome to Discover Pharma! Today is : January 20, 2026

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rare disease treatment

  • Clinical Development,Clinical Trials,Drug approval,Oncology,Pharmaceuticals and therapeutics,Rare Diseases,Regulatory Affairs

    FDA accepts Merck NDA for pimicotinib in tenosynovial giant cell tumor

    Merck has announced that the US Food and Drug Administration [...]

    January 13, 2026
  • Central Nervous System,Clinical Development,Clinical Trials,Neurosciences,Partnerships & Funding,Rare Diseases

    Servier acquires Kaerus drug candidate for Fragile X syndrome, the most common genetic cause of autism

    Servier has acquired KER-0193 from Kaerus Bioscience, gaining a potential [...]

    September 16, 2025
  • Cell & Gene Therapy,Regulatory Affairs

    UK experts urge Government to appoint Minister for cell and gene therapies

    Commission sets out 25 policy recommendations to unlock access and [...]

    June 23, 2025

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