Darling Dakota’s Life Taken By Rare Lysosomal Storage Disorder Called Wolman’s Disease

In October of 2009, Dakota was diagnosed with Wolman’s disease. Wolman’s disease belongs to a group of diseases known as Lysosomal Storage Disorders and is a rare genetic disorder caused by a deficiency of an enzyme known as lysosomal acid lipase (LAL or LIPA). This […]

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 […]