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rare diseases

  • Advanced Therapies,Biotech & Pharma Business,Clinical & Regulatory,Medical devices,Opinion & Events,Pharmaceuticals and therapeutics,Therapeutic Areas

    Terumo BCT expands access to rare disease therapies across Latin America

    Terumo Blood and Cell Technologies has announced regulatory approvals for [...]

    May 5, 2026
  • Clinical & Regulatory,Drug Discovery & Development,Therapeutic Areas

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

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

    May 5, 2026
  • Clinical & Regulatory,Drug Discovery & Development,Opinion & Events,Therapeutic Areas

    MHRA approves Biogen’s tofersen for ultra-rare genetic form of motor neurone disease

    The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has [...]

    May 5, 2026
  • Drug Discovery & Development,Therapeutic Areas

    Princess Royal opens new Insmed research facility in Cambridge focused on synthetic rescue

    Insmed has officially opened a new $10M, 17,000-square-foot research and [...]

    May 5, 2026
  • Advanced Therapies,Clinical & Regulatory,Therapeutic Areas

    Rare bone disorder: Novadip reports long-term success for pediatric patients treated under compassionate use programme

    Novadip Biosciences and Cliniques universitaires Saint-Luc have reported successful long-term [...]

    May 5, 2026
  • Advanced Therapies,Clinical & Regulatory,Therapeutic Areas

    First-in-class cell therapy for rare mitochondrial disorder gains FDA fast track

    FDA recognition accelerates development of first-in-class mitochondrial cell therapy for [...]

    May 5, 2026
  • Advanced Therapies,Biotech & Pharma Business,Clinical & Regulatory,Therapeutic Areas

    Ascidian boosts RNA therapy push with key hire and expert board for Stargardt trial

    Ascidian Therapeutics has appointed Murray Abramson as chief development officer. [...]

    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
  • Clinical & Regulatory,Drug Discovery & Development,Therapeutic Areas

    Nicotinamide riboside shows promise in rare premature aging disease

    Researchers in Japan have reported encouraging findings from the first [...]

    May 5, 2026
  • Advanced Therapies,Opinion & Events

    CAR T Vision coalition sets goal to double access to therapy by 2030

    A new international alliance, CAR T Vision, launched today with [...]

    May 5, 2026
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