Innovative Treatment of a Severe Disease

Promising Effect of High Dose Ambroxol Treatment on Neurocognition and Motor Development in a Patient With Neuropathic Gaucher Disease 2

Exploratory innovative treatments are often pursued and justified in diseases that are associated with fatal outcomes in early childhood. CENTOGENE is proud to have contributed to a pertinent success story in which a newborn with Gaucher disease type 2 developed largely normal after being prescribed an off-label treatment with a cough medication during the first three years of life. The case was reported in Frontiers in Neurology.


  • Guido Hartmann
  • et al.