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rare disease

  • Biotech & Pharma Business,Clinical & Regulatory,Pharmaceuticals and therapeutics,Research & Development,Therapeutic Areas

    BridgeBio gets FDA Priority Review for oral infigratinib in achondroplasia

    BridgeBio has secured FDA Priority Review for its oral infigratinib [...]

    October 7, 2026
  • Advanced Therapies,Biotech & Pharma Business,Clinical & Regulatory,Research & Development,Therapeutic Areas

    Novadip secures €10.4M to advance pivotal Phase 3 trial

    Novadip Biosciences has secured €10.4 million in convertible financing to [...]

    September 21, 2026
  • Advanced Therapies,Biotech & Pharma Business,Central Nervous System,Clinical & Regulatory,Manufacturing & Supply Chain,Pharmaceuticals and therapeutics,Research & Development,Therapeutic Areas

    Andelyn to manufacture FDA-approved Fayuvi gene therapy for Sanfilippo syndrome

    Andelyn Biosciences is manufacturing Ultragenyx’s newly FDA-approved Fayuvi gene therapy [...]

    September 21, 2026
  • Advanced Therapies,Biotech & Pharma Business,Central Nervous System,Clinical & Regulatory,Drug Discovery & Development,Pharmaceuticals and therapeutics,Research & Development,Therapeutic Areas

    NIH funds expanded access programme for Neurizon’s ALS therapy

    A US National Institutes of Health grant will fund an [...]

    September 16, 2026
  • Biotech & Pharma Business,Clinical & Regulatory,Drug Discovery & Development,Pharmaceuticals and therapeutics,Research & Development,Therapeutic Areas

    Sapience Therapeutics gets FDA clearance for ST316 FAP study

    The FDA has cleared Sapience Therapeutics to begin clinical development [...]

    September 16, 2026
  • Advanced Therapies,Clinical & Regulatory,Drug Discovery & Development,Pharmaceuticals and therapeutics,Research & Development

    Genespire starts international MMA study to support GENE202 development

    Genespire has started enrolling children with severe methylmalonic acidemia in [...]

    August 25, 2026
  • Clinical & Regulatory,Drug Delivery & Formulation,Pharmaceuticals and therapeutics,Research & Development,Therapeutic Areas

    FDA grants Priority Review to Belite Bio’s tinlarebant for Stargardt disease

    The FDA has accepted Belite Bio’s application for tinlarebant and [...]

    August 13, 2026
  • Biotech & Pharma Business,Clinical & Regulatory,Drug Discovery & Development,Pharmaceuticals and therapeutics,Research & Development,Therapeutic Areas

    Vaderis raises $152M and starts Phase 3 trial of engasertib in HHT

    Vaderis Therapeutics has raised $152M and started a global Phase [...]

    August 12, 2026
  • Central Nervous System,Clinical & Regulatory,Research & Development,Therapeutic Areas

    Zilucoplan accepted for eligible myasthenia gravis patients in NHS Scotland

    Zilucoplan has been accepted for restricted use in NHS Scotland [...]

    August 11, 2026
  • Clinical & Regulatory,Pharmaceuticals and therapeutics,Research & Development

    Grifols completes Phase 3 SPARTA study of Prolastin-C in alpha-1 emphysema

    Grifols has completed patient follow-up in its Phase 3 SPARTA [...]

    August 11, 2026
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recent posts
  • Novadip secures €10.4M to advance pivotal Phase 3 trial
    Categories: Advanced Therapies, Biotech & Pharma Business, Clinical & Regulatory, Research & Development, Therapeutic Areas
  • Telix to acquire ITM in $1.65BN radiopharmaceutical deal
    Categories: Biotech & Pharma Business, Manufacturing & Supply Chain, Pharmaceuticals and therapeutics, Research & Development, Therapeutic Areas
  • Andelyn to manufacture FDA-approved Fayuvi gene therapy for Sanfilippo syndrome
    Categories: Advanced Therapies, Biotech & Pharma Business, Central Nervous System, Clinical & Regulatory, Manufacturing & Supply Chain, Pharmaceuticals and therapeutics, Research & Development, Therapeutic Areas
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