Floridians Recognized for Role in Helping Rare Disease Patients Who Need Lifesaving Therapies
ANNAPOLIS, Md., Sept. 1, 2011 /PRNewswire/ -- This month, Floridians are recognizing "Plasma Protein Therapies Month," by raising awareness for the valuable contributions of plasma donors throughout the "Sunshine State" and for the rare, genetic diseases treated with the therapies that are made possible through plasma donation.
Plasma protein therapies, which include plasma-derived therapies and recombinant blood clotting factors (a biotechnology product), are used every day to treat people with bleeding disorders, such as hemophilia, that causes painful internal bleeding and debilitating joint damage; primary immunodeficiency diseases, which prevent a person from fighting off even common infections; and alpha-1 antitrypsin deficiency, also known as genetic chronic obstructive pulmonary disease (COPD), a disease that severely damages the liver and lungs. In addition, a plasma protein therapy, albumin, is used in critical care settings, when treating severe trauma, burns and during major surgery.
"It has given Dominik a normal childhood," said Kim Culver of her 10-year-old son who has a primary immune deficiency (PID) and who injects a plasma protein therapy, immune globulin, regularly from home. The Fort Myers mother says, "I can't describe the difference from before he was taking the therapy and after." Kim describes a life spent in doctors' offices, hospitals and traveling to specialists for four years before Dominik was diagnosed. Now Kim says he lives a happy, normal life and is pursuing a black belt in Tae Kwon Do, something that seemed unthinkable just a few years ago. Not only does Dominik have a PID, but also he has been diagnosed with von Willebrand disease, an inherited bleeding disorder that affects the body's ability to clot and that requires a different type of plasma protein therapy to treat. Kim routinely travels with her son three hours to a see a hematologist in Miami, who has never seen a case like Dominik. Kim looks forward to vis