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achondroplasia

  • Clinical & Regulatory,Pharmaceuticals and therapeutics,Research & Development

    BridgeBio Phase 3 achondroplasia trial published in NEJM

    BridgeBio has published results from its pivotal Phase 3 Propel [...]

    June 30, 2026
  • Therapeutic Areas

    Rare Disease Day 2026: Er-Kim pushes to close access gap in underserved markets

    As Rare Disease Day 2026 highlights the challenges faced by [...]

    May 5, 2026
  • Clinical & Regulatory,Pharmaceuticals and therapeutics

    MHRA grants Promising Innovative Medicine designation to TransCon CNP for achondroplasia

    Ascendis Pharma UK Limited announced that TransCon CNP (navepegritide) has [...]

    May 5, 2026
  • Clinical & Regulatory,Drug Discovery & Development,Pharmaceuticals and therapeutics,Therapeutic Areas

    A potential first-in-class treatment for children with achondroplasia is now in clinical testing

    Tyra Biosciences has dosed the first child in BEACH301, its [...]

    May 5, 2026

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