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Welcome to Discover Pharma! Today is : September 19, 2026

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lysosomal storage disorders

  • Clinical & Regulatory,Drug Discovery & Development,Pharmaceuticals and therapeutics,Research & Development,Therapeutic Areas

    Polaryx advances Phase 2 trial of PLX-200 in four rare diseases

    Polaryx Therapeutics is preparing to start its Phase 2 SOTERIA [...]

    August 26, 2026
  • Advanced Therapies,Drug Discovery & Development,Manufacturing & Supply Chain,Therapeutic Areas

    Andelyn and Queen’s University partner on AAV9 gene therapy for GM2 gangliosidoses

    Andelyn Biosciences and Queen’s University have partnered to develop and [...]

    August 19, 2026
  • Biotech & Pharma Business,Opinion & Events,Therapeutic Areas

    Chiesi launches second Find For Rare research grants programme at ERA 2026

    Chiesi Global Rare Diseases has launched the second edition of [...]

    June 9, 2026
  • Biotech & Pharma Business,Clinical & Regulatory,Drug Discovery & Development,Pharmaceuticals and therapeutics,Therapeutic Areas

    Polaryx therapeutics selects contract research organization for SOTERIA phase 2 basket trial

    Polaryx Therapeutics, Inc has selected a leading contract research organization [...]

    May 5, 2026
  • Biotech & Pharma Business,Therapeutic Areas

    Chiesi and Key2Brain extend global partnership to advance blood-brain barrier-crossing therapies for ultra-rare diseases

    Chiesi Group has signed a worldwide license agreement with Swedish [...]

    May 5, 2026

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