Clinical Development Program in LAL Deficiency By Synageva


LAL Deficiency, a lysosomal storage disorder, has early and late onset phenotypes, also referred to as Wolman Disease or Cholesteryl Ester Storage Disease (CESD), respectively. There is no approved treatment for LAL Deficiency.  Interventional research studies are currently recruiting patients for treatment with SBC-102.  SBC-102 […]