by Peter Ciszewski | Aug 26, 2018
Gail Cawkwell, MD, PhD, Senior Vice President, Medical Affairs at Intercept, provides discusses some of the physician and patient resources regarding primary biliary cholangitis (PBC), a rare liver disease that is caused by an autoimmune reaction. When bile is...
by Peter Ciszewski | Aug 26, 2018
The U.S. Food and Drug Administration (FDA) approved lanadelumab (Takhzyro), the first monoclonal antibody to help prevent attacks of hereditary angioedema (HAE) in patients aged 12 years and older with types I and II of the disease. HAE is a rare and potentially...
by Peter Ciszewski | Aug 25, 2018
Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals, provides an overview of cystinosis, an inherited condition in which the body accumulates the amino acid cystine (a building block of proteins) within the...
by Peter Ciszewski | Aug 24, 2018
Overview Acute myeloid leukemia (AML) is a cancer that affects the blood and bone marrow. Conditions are generally called “acute” when they develop quickly and have an aggressive course. The signs and symptoms of AML vary but may include easy bruising;...
by Peter Ciszewski | Aug 23, 2018
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City, discusses her experience working with rare diseases and neurogenetics.
