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Rare Diseases

  • Biologics & Biosimilars,Global health,Market Access & Commercialization,Pharmaceuticals and therapeutics,Rare Diseases,Supply Chain & Logistics

    Er-Kim to commercialize Nulibry and Zokinvy across Central and Eastern Europe, Mediterranean and Eurasia

    Er-Kim has signed an agreement with Sentynl Therapeutics to distribute [...]

    September 29, 2025
  • Biologics & Biosimilars,Biotech,Partnerships & Funding,Rare Diseases,Research & Development

    DEBRA Research and Nonsense Therapeutics launch ReadOn Therapeutics to target epidermolysis bullosa

    DEBRA Research gGmbH, a global non-profit dedicated to advancing research [...]

    September 26, 2025
  • Clinical Development,Clinical studies,Patient Centricity,Rare Diseases

    CureDuchenne announces Fuerza 2025 conference in Mexico City on Duchenne and Becker muscular dystrophy

    CureDuchenne will host CureDuchenne Fuerza 2025, a one-day educational and [...]

    September 24, 2025
  • Biologics & Biosimilars,Clinical Trials,Oncology,Pharmaceuticals and therapeutics,Rare Diseases,Research & Development

    Poolbeg secures free supply of bispecific antibody for POLB 001 trial at The Christie

    Poolbeg Pharma has appointed Accelerating Clinical Trials (ACT) to run [...]

    September 25, 2025
  • 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
  • Drug Development,Neurosciences,Rare Diseases,Regulatory Affairs

    SOM Biotech secures EMA orphan designation opinion for Huntington’s drug SOM3355

    Barcelona-based biotech advances Huntington’s programme as EMA backs orphan designation, [...]

    September 15, 2025
  • Clinical Development,Clinical Trials,Oncology,Pharmaceuticals and therapeutics,Rare Diseases,Regulatory Affairs

    FDA and Telix agree resubmission plan for Pixclara glioma imaging agent

    Telix Pharmaceuticals has reached agreement with the US Food and [...]

    September 14, 2025
  • Drug Development,Movers & Shakers,Oncology,Rare Diseases,Regulatory Affairs

    Amorphical names Eden Ben CEO to drive regulatory strategy and expand nano-mineral therapeutics pipeline

    Amorphical has appointed Eden Ben as chief executive officer, as [...]

    September 10, 2025
  • Clinical Development,Neurosciences,Partnerships & Funding,Pharmaceuticals and therapeutics,Rare Diseases,Research & Development

    NRG Therapeutics closes oversubscribed £50m series B to advance clinical data in Parkinson’s and proof of concept in ALS/MND

    NRG Therapeutics Ltd, a neuroscience company developing treatments targeting mitochondrial [...]

    September 8, 2025
  • Clinical Trials,Oncology,Rare Diseases,Regulatory Affairs

    Protagonist Therapeutics gains FDA Breakthrough Therapy designation for rusfertide in polycythemia vera

    Protagonist Therapeutics has announced that the US Food and Drug [...]

    August 27, 2025
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