Emerging therapeutic targets for Gaucher disease.

  title={Emerging therapeutic targets for Gaucher disease.},
  author={Einat B. Vitner and Ayelet Vardi and Timothy Martin Cox and Anthony H Futerman},
  journal={Expert opinion on therapeutic targets},
  volume={19 3},
INTRODUCTION Gaucher disease (GD) is an inherited metabolic disorder caused by mutations in the glucocerebrosidase (GBA1) gene. Although infusions of recombinant GBA ameliorate the systemic effects of GD, this therapy has no effect on the neurological manifestations. Patients with the neuronopathic forms of GD (nGD) are often severely disabled and die prematurely. The search for innovative drugs is thus urgent for the neuronopathic forms. AREAS COVERED Here we briefly summarize the available… CONTINUE READING
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