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Rare Diseases

  • Biologics & Biosimilars,Clinical Development,Clinical Trials,Drug Development,Movers & Shakers,Orthapaedics,Rare Diseases

    Levicept appoints James Sandy as chief development officer to advance LEVI-04 for osteoarthritis

    Levicept has strengthened its executive team with the appointment of [...]

    February 4, 2026
  • Cardiovascular diseases,Clinical Trials,Rare Diseases,Research & Development

    Cereno Scientific completes expanded access program milestone for CS1 in pulmonary arterial hypertension

    Cereno Scientific has completed the last patient’s last visit in [...]

    February 4, 2026
  • Clinical Development,Drug approval,Genetic Diseases,Rare Diseases,Regulatory Affairs

    Pegunigalsidase alfa every four weeks dosing backed by CHMP for adult Fabry patients

    The European Medicines Agency’s Committee for Medicinal Product for Human [...]

    February 2, 2026
  • Clinical Development,Clinical Trials,Pharmaceuticals and therapeutics,Rare Diseases

    Boehringer Ingelheim’s apecotrep reduces proteinuria in Phase 2 FSGS trial

    Boehringer Ingelheim has reported Phase 2 clinical trial results showing [...]

    January 30, 2026
  • Central Nervous System,Clinical Trials,Drug Development,Rare Diseases,Research & Development

    Skyhawk Therapeutics reports positive nine-month interim results for SKY-0515 in Huntington’s disease

    Skyhawk Therapeutics has reported positive nine-month interim results from its [...]

    January 29, 2026
  • Biologics & Biosimilars,Cell & Gene Therapy,Clinical Development,Drug Development,Rare Diseases,Research & Development

    iXCells Biotechnologies partners with Rosebud Biosciences to advance organoid models for rare diseases

    iXCells Biotechnologies and Rosebud Biosciences have announced a partnership to [...]

    January 29, 2026
  • Drug approval,Immunology,Pharmaceuticals and therapeutics,Rare Diseases,Regulatory Affairs,RNA Therapeutics

    Donidalorsen granted EU approval for hereditary angioedema prevention

    Donidalorsen has received European Union marketing authorisation for the routine [...]

    January 23, 2026
  • Biotech,Drug Development,Partnerships & Funding,Rare Diseases,Technology and platforms

    Mendra launches with $82 million Series A to develop rare disease therapies

    Mendra, Inc. has launched with the closing of an $82 [...]

    January 23, 2026
  • Biotech,Central Nervous System,Clinical Development,Clinical Trials,Pharmaceuticals and therapeutics,Rare Diseases

    Exciva raises €51 million Series B to fund phase 2 Alzheimer’s agitation trial

    Exciva GmbH has raised €51 million ($59m) in a Series [...]

    January 20, 2026
  • Biotech,Clinical Development,Clinical studies,Rare Diseases,Research & Development

    DEBRA Research and University Medical Center Groningen launch dedicated clinical trial site for Epidermolysis Bullosa

    DEBRA Research and the University Medical Center Groningen (UMCG) have [...]

    January 19, 2026
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