This approach is called substrate reduction therapy.Eliglustat (Cerdelga) (approved in 2014)  is also a small molecule. The compound is believed to work by inhibition of glucosylceramide synthase. Epidemiology
The National Gaucher Foundation (United States) states the incidence of Gauchers disease is about one in 20,000 live births. Around one in 100 people in the general US population is a carrier for type I Gauchers disease, giving a prevalence of one in 40,000. Among Ashkenazi Jews, the rate of carriers is considerably higher, at roughly one in 15.Type II Gauchers disease shows no particular preference for any ethnic group.Type III Gauchers disease is especially common in the population of the northern Swedish region of Norrbotten, where the incidence of the disease is one in 50,000. History
The disease was first recognized by the French doctor Philippe Gaucher, who originally described it in 1882 and lent his name to the condition. In 1902, its mode of inheritance was discovered by Nathan Brill. The neuronal damage associated with the disease was discovered in the 1920s, and the biochemical basis for the disease was elucidated in the 1960s by Roscoe Brady. The first effective treatment for the disease, the drug alglucerase (Ceredase), was approved by the FDA in April 1991. An improved drug, imiglucerase (Cerezyme), was approved by the FDA in May 1994 and has replaced the use of Ceredase. October is National Gauchers Disease Awareness Month in the United States.
Prominent people with disease
Wallace Chapman; New Zealand radio and television personality
Anne Begg; Scottish politician
Adam Rose; American Actor

Gallery
See also
References
External links

Gaucher Disease at NINDS