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rare diseases

  • 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
  • Clinical & Regulatory,Drug Delivery & Formulation

    FDA approves Khindivi oral solution for pediatric adrenal insufficiency

    Eton Pharmaceuticals has received US regulatory approval for Khindivi, the [...]

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

    Patient priorities must shape trial design, says One2Treat lead

    To mark Clinical Trials Day, Discover Pharma speaks with Tom [...]

    May 5, 2026
  • Advanced Therapies,Clinical & Regulatory,Drug Discovery & Development

    Why rare disease trials are poised to transform drug development by 2030

    Today on Clinical Trials Day, we spotlight the evolving landscape [...]

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

    FTD trial milestone reached as Vesper Bio enrols first asymptomatic GRN carriers

    Vesper Bio has reached a key enrolment milestone in its [...]

    May 5, 2026
  • Advanced Therapies,Biotech & Pharma Business

    Creyon Bio and Lilly collaborate on AI-designed RNA-targeted oligo therapies

    Partnership aims to accelerate development using computational drug design Creyon [...]

    May 5, 2026
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