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Rare Diseases

  • Biotech,Cell & Gene Therapy,Clinical Development,Clinical Trials,Opthalmology,Rare Diseases,Research & Development

    AAVantgarde completes enrollment in LUCE-1 Phase 1 and Phase 2 trial of gene therapy AAVB-081 for Usher syndrome type 1B

    AAVantgarde Bio has completed enrollment in LUCE-1, a first-in-human Phase [...]

    January 16, 2026
  • Biotech,Clinical Development,Clinical studies,Clinical Trials,Healthcare leadership,Movers & Shakers,Rare Diseases

    Veristat names Kim McLean Boericke as CEO to drive next phase of clinical research growtheristat names Kim McLean Boericke as CEO to drive next phase of clinical research growth

    Veristat, a global clinical research organization (CRO) and consultancy specializing [...]

    January 15, 2026
  • Biotech,Rare Diseases,Regulatory Affairs,Research & Development

    Minovia secures two new US patents for mitochondrial augmentation therapy platform

    Minovia Therapeutics has been granted two new US patents covering [...]

    January 13, 2026
  • Biotech,Cell & Gene Therapy,Genetic Diseases,Genomics and sequencing,Rare Diseases,Research & Development,Technology and platforms

    PacBio HiFi adopted to study sudden unexplained death in childhood and advance precision therapies for rare diseases

    PacBio is supporting two research initiatives that use its HiFi [...]

    January 13, 2026
  • Clinical Development,Clinical Trials,Drug approval,Oncology,Pharmaceuticals and therapeutics,Rare Diseases,Regulatory Affairs

    FDA accepts Merck NDA for pimicotinib in tenosynovial giant cell tumor

    Merck has announced that the US Food and Drug Administration [...]

    January 13, 2026
  • Drug Development,Market Access & Commercialization,Opthalmology,Pharmaceuticals and therapeutics,Rare Diseases

    Age-related macular degeneration market across 7MM forecast to reach $20.5 billion by 2034

    The age-related macular degeneration market across the seven major markets [...]

    December 29, 2025
  • Clinical Trials,Neurosciences,Rare Diseases

    UCB reports positive phase 3 Gemz results as fenfluramine cuts seizure frequency in CDKL5 deficiency disorder

    UCB has reported positive findings from the Gemz phase 3 [...]

    December 10, 2025
  • 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
  • Biologics & Biosimilars,Drug approval,European biotech,Hematology,Rare Diseases

    Grifols’ Biotest secures first approval for fibrinogen concentrate in Germany

    Grifols’ Biotest has received its first regulatory approval for a [...]

    November 14, 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
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