PFIC Patient Describes Their Treatment Options скачать в хорошем качестве

PFIC Patient Describes Their Treatment Options

Kealey-Shay Spolijarevic has progressive familial intrahepatic cholestasis (PFIC), a rare hereditary disease in which an individual is unable to properly remove bile acids from their liver. She describes a few of the treatment options available for PFIC patients. PFIC, according to the NIH, is estimated to affect between one in every 50,000 to 100,000 children born worldwide and causes progressive life-threatening liver disease. The most prominent and problematic ongoing manifestation of PFIC is pruritus, which often results in a severely diminished quality of life. In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life, and nearly all individuals with PFIC will require treatment before age 30. As Kealey-Shay describes, there are several treatments available for PFIC, some of which include ursodiol, cholestyramine, rifampicin, and naloxone. One issue she brings up is the potential for these medications to work for a while and then stop working, thereby making finding a long-term solution difficult. For more information on PFIC and other rare diseases, sign up for our newsletter at https://checkrare.com/sign-up-for-our...