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Welcome to Discover Pharma! Today is : December 6, 2025

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rare diseases

  • Orphan drugs,Rare Diseases,Regulatory Affairs

    EMA grants orphan drug designation to Kedrion treatment for congenital aceruloplasminemia

    Kedrion has received orphan drug designation from the European Medicines [...]

    November 27, 2025
  • Neurosciences,Rare Diseases,Research & Development

    Successful treatment for ALS could deliver billions in global value

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

    November 13, 2025
  • Biologics & Biosimilars,Clinical Development,European biotech,Global health,Hematology,Rare Diseases,Technology and platforms

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

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

    October 10, 2025
  • Biotech,Genomics and sequencing,Partnerships & Funding,Rare Diseases,Research & Development

    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 [...]

    October 7, 2025
  • Biotech,Clinical Development,Clinical Trials,Drug Development,Global health,Partnerships & Funding,Rare Diseases,Research & Development

    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 [...]

    October 1, 2025
  • 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,Chronic diseases,Clinical Development,Clinical Trials,Global health,Market Access & Commercialization,Medical devices,Patient Centricity,Pharmaceuticals and therapeutics,Regulatory Affairs

    Terumo BCT expands access to rare disease therapies across Latin America

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

    September 22, 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
  • Drug Development,Patient Centricity,Rare Diseases,Regulatory Affairs

    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 [...]

    July 28, 2025
  • Rare Diseases,Research & Development

    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 [...]

    July 22, 2025
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