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rare diseases

  • Drug Discovery & Development,Therapeutic Areas

    Successful treatment for ALS could deliver billions in global value

    New research commissioned by Challenge Works and conducted by the [...]

    May 5, 2026
  • Advanced Therapies,Biotech & Pharma Business,Clinical & Regulatory,Drug Discovery & Development,Opinion & Events,Therapeutic Areas

    Terumo BCT and Hemex Health join forces to improve outcomes for sickle cell patients

    Terumo Blood and Cell Technologies (Terumo BCT), a global medical [...]

    May 5, 2026
  • Biotech & Pharma Business,Drug Discovery & Development,Genomics and sequencing,Therapeutic Areas

    Chiesi and Arbor partner on rare disease gene editing programmes targeting PH1 and other liver disorders

    Chiesi Group has entered into an exclusive global partnership with [...]

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

    SeaBeLife secures €2 million to advance dual-targeted therapies for dry AMD and severe acute hepatitis

    SeaBeLife, a biotechnology company developing novel drug candidates to block [...]

    May 5, 2026
  • Advanced Therapies,Biotech & Pharma Business,Manufacturing & Supply Chain,Opinion & Events,Pharmaceuticals and therapeutics,Therapeutic Areas

    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 [...]

    May 5, 2026
  • 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
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