Rare Disease

Brand Name
Description
Ocaliva®
(obeticholic acid)
OCALIVA is indicated for the treatment of primary biliary cholangitis in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA
Brand Name
Description
Brand Name
Description
Brand Name
Description
Orladeyo™
(berotralstat)
Orladeyo™ is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older
Diseases
Description
Alagille
syndrome
Alagille syndrome is a genetic syndrome that can affect the liver and other parts of the body. The liver problems result from having fewer small bile ducts than normal in the liver. This leads to bile building-up inside the liver, which in turn causes liver scarring and damage. For more information on available treatment options, please contact us on [email protected]
Generalized
Lipodystrophy
Generalized lipodystrophy (GL) is a rare condition that results in having little to no fat tissue all over the body, which affects the individual’s appearance. The lack of body fat results in a loss of leptin, an important hormone that helps the body function properly, which can lead to health problems such as high triglycerides and high blood sugar. For more information on available treatment options, please contact us on [email protected]
Hereditary
Angioedema
Hereditary Angioedema (HAE), is a very rare and potentially life-threatening genetic condition characterized by recurrent attacks of severe swelling in various parts of the body, including the hands, feet, genitals, stomach, face and/or throat. For more information on available treatment options, please contact us on [email protected]
Homozygous Familial
Hypercholesterolaemia
Homozygous familial hypercholesterolaemia (HoFH) is a rare life-threatening condition characterized by markedly elevated circulating levels of low-density lipoprotein cholesterol (LDL-C) and accelerated, premature atherosclerotic cardiovascular disease (ACVD). For more information on available treatment options, please contact us on [email protected]
Brand Name
Description
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