Programme SOM0208 for the Treatment of Niemann–Pick Type C Disease
SOM0208 is a small molecule for the treatment of Niemann–Pick C disease.
Niemann–Pick type C is a lysosomal storage disorder caused by defects in the intracellular transport and metabolism of cholesterol and glycolipids. The disease has a broad clinical spectrum. Affected individuals may have enlargement of the spleen (splenomegaly) and liver (hepatomegaly), or enlarged spleen or liver combined (hepatosplenomegaly), but this finding may be absent in later onset cases. Prolonged jaundice or elevated bilirubin can present at birth.
