Sphingomyelinase Deficiency Medication

Updated: Aug 18, 2016
  • Author: Lynne Ierardi-Curto, MD, PhD; Chief Editor: Luis O Rohena, MD, FAAP, FACMG  more...
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Medication Summary

Therapy using medications currently is not a component of the standard of care in patients with Niemann-Pick disease (NPD). A phase 1b clinical trial to evaluate the efficacy of recombinant human acid sphingomyelinase for the treatment of the non-neurologic manifestations of adults with NPD type B will soon be initiated (see Genzyme Therapeutics).