Nonalcoholic steatohepatitis (NASH)

  • Nonalcoholic steatohepatitis or NASH is a common liver disease. It resembles alcoholic liver disease but occurs in people who drink little or no alcohol.
  • The pathophysiology involves fat accumulation (steatosis), inflammation, and, variably, fibrosis. Steatosis results from hepatic triglyceride accumulation.
  • Most patients are asymptomatic. However, some have fatigue, malaise, or right upper quadrant abdominal discomfort.
  • The only widely accepted treatment goal is to eliminate potential causes and risk factors.
  • NASH (sometimes called steatonecrosis) is diagnosed most often in patients between 40 and 60 years but can occur in all age groups.
  • The true prevalence of NASH also remains elusive, mostly because of the lack of a definitive laboratory test.


Primary sclerosing cholangitis:

  • Primary sclerosing cholangitis (PSC) is a disease of the bile ducts that causes inflammation and obliterative fibrosis of bile ducts inside and/or outside of the liver.
  • Etiology and pathogenesis of PSC still pose many unresolved questions and remain a scientific and clinical challenge.
  • There is a 2-3:1 male-to-female predilection in primary sclerosing cholangitis.
  • Approximately 75% of individuals with PSC also have inflammatory bowel disease (IBD).
  • The only definitive treatment for PSC is liver transplantation. No drug has yet been approved for PSC.
  • Orphan indication with annual incidence of 0.068–1.3 per 100,000 people.


Systemic Sclerosis (SSc)

  • Systemic sclerosis is an autoimmune, connective tissue disease.

There is no known clear cause for scleroderma and systemic sclerosis which is characterized by the thickening of the skin caused by accumulation of collagen, and by injuries to the smallest arteries.

  • There are two overlapping forms:
    • Limited cutaneous scleroderma.
    • Diffuse cutaneous scleroderma covers more of the skin, and is at risk of progressing to the visceral organs, including the kidneys, heart, lungs and gastrointestinal tract.
    • Survival is determined by the severity of visceral disease.
    • Patients with limited cutaneous scleroderma have a good prognosis, with 75% reaching a 10-year survival period.
    • Patients with diffuse cutaneous scleroderma have a 10-year survival rate of 55%.
    • Affects about 100,000 in the U.S (eligible for orphan and fast track status).