Logo
  • About us
    • Story and Mission
    • Board of Directors
    • Leadership Team
    • Investors
  • Our Science
    • Lysosomal Storage Disorders
    • Our Lead Programme
  • Azafaros for Patients
    • Disease information
    • Our Therapeutic Approach
    • Patient Resources and Organizations
    • Expanded Access Policy
    • Patient stories
  • News & Media
  • Events
  • Contact
  • Clinical Studies

  • Home
  • About us
    • Story and Mission
    • Board of Directors
    • Leadership Team
    • Investors
  • Our Science
    • Lysosomal Storage Disorders
    • Our Lead Programme
  • Azafaros for Patients
    • Disease information
    • Our Therapeutic Approach
    • Patient Resources and Organizations
    • Expanded Access Policy
    • Patient stories
  • News & Media
  • Events
  • Contact
  • Clinical Studies
  • Other pages
    • Imprint
    • Navigate Cross-Border Referral

  • News & Media
    • Azafaros Completes Enrollment of Phase 3 (NAVIGATE) Study Evaluating Nizubaglustat for the Treatment of Patients with GM1/GM2 Gangliosidoses

    • News
      • Azafaros Announces Publication of Phase 2 RAINBOW Study Data for Nizubaglustat in Molecular Genetics and Metabolism Journal
      • Azafaros strengthens leadership team with appointment of Amy Sullivan as Chief Financial Officer
      • Azafaros Announces Publication of Preclinical Efficacy Data with Nizubaglustat in GM2 Gangliosidosis
      • Azafaros Announces Key Scientific Oral and Poster Presentations on Nizubaglustat Accepted for WORLDSymposium™ 2026
      • Azafaros to Present at J.P. Morgan’s 44th Annual Healthcare Conference
      • Azafaros further strengthens its Board of Directors with appointment of biotech entrepreneur, Dr. Thierry Abribat
      • Azafaros Announces Initiation of two Global Phase 3 studies with Nizubaglustat in Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses, respectively
      • Azafaros to Present at BIO International Convention 2025 Following Successful €132M Series B Financing
      • Azafaros Secures € 132M in Oversubscribed Series B Financing to advance Phase 3 clinical programs of innovative therapies in lysosomal storage disorders
      • Azafaros to Present at J.P. Morgan’s 43rd Annual Healthcare Conference
      • Azafaros granted important regulatory designations and clearance by European authorities for global Phase 3 studies, to be initiated in 2025
      • Azafaros Announces Key Scientific Oral and Poster Presentations on Nizubaglustat Accepted for WORLDSymposium™ 2025
      • Positive Niemann-Pick disease type C (NPC) and GM2 gangliosidosis data from nizubaglustat Phase 2 RAINBOW study conducted by Azafaros presented at major metabolic disease conference
      • Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C
      • Azafaros announces promotion of Dr Anke Arnold-Tugulu to Chief Regulatory Officer
      • Azafaros announces completion of 12-week Phase 2 RAINBOW study evaluating lead asset nizubaglustat in rare disease patients
      • Azafaros to present data from PRONTO study in patients with GM1 and GM2 gangliosidoses at the 20th annual WORLDSymposium
      • Azafaros’ Phase 2 RAINBOW study, evaluating nizubaglustat in GM2 and NPC patients, is now fully enrolled
      • Azafaros Announces Enrollment of First Patient in Phase 2 RAINBOW Study Evaluating AZ-3102 in GM2 and NP-C Patients
      • Azafaros Appoints Chief Operating Officer and Head of Finance
      • Beyond Biotech podcast 35: Rare Disease Day
      • Azafaros Receives Additional Regulatory Designations for AZ-3102 from FDA, EMA and MHRA
      • Azafaros Strengthens Medical and Scientific Management Team Reflecting Clinical Progress
      • BREAKING NEWS: Azafaros Receives FDA’s IND Clearance and Fast Track Designation
      • Azafaros Announces FDA Grant of Orphan Drug Designation for AZ-3102 in the Treatment of Niemann-Pick Disease
      • Azafaros Presents Positive Clinical and Preclinical Data Supporting Development of Lead Compound AZ-3102 in Lysosomal Storage Disorders at the 18th Annual WORLDSymposium™ Conference
      • Azafaros Receives FDA Orphan Drug Designation for AZ-3102 in GM2 Gangliosidosis
      • Azafaros Announces Multiple Presentations at 18th Annual WORLDSymposium™
      • Azafaros Appoints Stefano Portolano, M.D., as Chief Executive Officer
      • Azafaros enters clinical stage with AZ-3102, an oral small molecule being developed for rare neurogenetic disorders
      • Azafaros expands executive management team and operations
      • Azafaros completes EUR 25 million Series A financing to advance rare metabolic disorders pipeline
      • Azafaros B.V. secures seed funding to develop new treatments in rare metabolic disorders
      • Azafaros to Present at BIO International Convention 2026
    • Media
      • Podcast: Stefano Portolano on Nizubaglustat and the Future of Rare Disease Treatment
      • Patient Family Documentary: A Father’s Search for Hope in Brazil for His Son, Facundo, Living with Tay-Sachs Disease (GM2)
  • Clinical Studies
    • First-In-Human Study
    • RAINBOW Study
    • PRONTO Study
    • NAVIGATE Study
    • PRISMA Study

    • Sitemap
    • Privacy Policy
    • Cookie declaration

    Need more information? Contact us.
    Get in touch
    About us

    Our Story and Mission
    Our Board of Directors
    Our Leadership Team
    Our Investors

    Our Science

    Lysosomal Storage Disorders
    Our Lead Programme

    Links

    Privacy Policy
    Imprint

    Contact

    info@azafaros.com

    Office

    Gooimeer 2-35
    1411 DC Naarden
    The Netherlands

    Sitemap | Privacy Policy | Cookie declaration | Website by CM Specialist
    2026 @ Azafaros B.V.