Krouzon Drug Development Program:

Pediatric Rare Diseases

Krouzon is developing pharmacological treatments for FGFR2-related Syndromic Craniosynostosis.

Syndromic Craniosynostosis

Unmet Medical Need

Syndromic Craniosynostosis is an unmet medical need, and over 80% of them are caused by mutations in Fibroblast Growth Factor Receptor 2 (FGFR2) gene.

Krouzon Pharmaceuticals is developing pharmacological treatments for the following most common FGFR2-related Syndromic Craniosynostosis:

  • Pfeiffer syndrome

  • Apert syndrome

  • Crouzon syndrome

The developmental disorders of Syndromic Craniosynostosis include anatomical, physiological, neurological, and psychological disorders as described below:

  1. ANATOMICAL DISORDERS:

    • Severe Deformity of head and face

  2. PHYSIOLOGICAL AND NEUROLOGICAL DISORDERS:

    • Developmental delay

    • Vision disorders

    • Hearing loss (70% cases)

    • Difficulty in breathing

    • Sleep apnea

    • Gastrointestinal disorders

    • Precocious puberty

    • Seizures

    • Death in severe cases

  3. PSYCHOLOGICAL IMPACT:

    • Children experience lifelong social isolation