Upcoming Events at NIH Building 45
Description
The Food and Drug Administration (FDA) is announcing the following public workshop entitled “Developing Alpha-1 Antitrypsin Therapeutics.”
Event Details
The Food and Drug Administration (FDA) is announcing the following public workshop entitled “Developing Alpha-1 Antitrypsin Therapeutics.”
Purpose of the workshop:
The purpose of the public workshop is to foster the development of therapeutic products for the treatment of alpha-1 antitrypsin deficiency (AAT deficiency).
Background:
AAT deficiency is a hereditary disorder that can be associated with the development of lung and liver disease. The age at which individuals initially present with the signs and symptoms of AAT deficiency can vary, but individuals often present between 20 and 50 years of age. Symptoms of lung disease are more common than liver disease and may include wheezing, shortness of breath with activity, and reduced exercise tolerance. Ultimately, emphysema (the state of irreversible damage to and partial loss of the small air sacs of the lung) may occur. The symptoms of liver disease may include yellowing of the skin and the whites of the eyes (jaundice), and, if more advanced, swelling of the abdomen due to cirrhosis (liver damage).
Purpose of the workshop:
The purpose of the public workshop is to foster the development of therapeutic products for the treatment of alpha-1 antitrypsin deficiency (AAT deficiency).
Background:
AAT deficiency is a hereditary disorder that can be associated with the development of lung and liver disease. The age at which individuals initially present with the signs and symptoms of AAT deficiency can vary, but individuals often present between 20 and 50 years of age. Symptoms of lung disease are more common than liver disease and may include wheezing, shortness of breath with activity, and reduced exercise tolerance. Ultimately, emphysema (the state of irreversible damage to and partial loss of the small air sacs of the lung) may occur. The symptoms of liver disease may include yellowing of the skin and the whites of the eyes (jaundice), and, if more advanced, swelling of the abdomen due to cirrhosis (liver damage).
