A Portacath or PICC Line Attracts Young Alpha-1 Patients
I knew some alpha-1 antitrypsin deficiency (AATD) patients have Portacaths and now I understand why.
Hepatologists
Assessing Infant Malnutrition Secondary to Chronic Cholestatic Liver Disease
We explore the importance of assessing the nutritional status of infants diagnosed with a chronic cholestatic liver disease.
Preparing to Care for My Son After His Alagille Syndrome Diagnosis
After we received the diagnosis of Alagille syndrome (ALGS) for my son, we were thrown into a new world of managing multiple medications.
Test Your Knowledge About Genetics in Alagille Syndrome
Take our quick quiz on the genetics and mutations involved in Alagille syndrome (ALGS) and how they are expressed in the disease.
New Cholangiocarcinoma Noncoding RNA Could Become an Intervention Target
Proteasome 20S subunit alpha 3 antisense RNA 1 (PSMA3-AS1) represents a new cancer-promoting long noncoding RNA in cholangiocarcinoma, as published in Aging. This finding suggests that PSMA3-AS1 could be a target for the treatment of the disease. It is already known that long noncoding RNAs including PSMA3-AS1 play a crucial role in the development and…
Postoperative CA19-9 Shows Prognostic Significance in Cholangiocarcinoma
Researchers concluded that the levels of the carbohydrate antigen 19-9 (CA19-9) had a better prognostic performance in patients with intrahepatic cholangiocarcinoma when measured postoperatively, as published in Frontiers in Oncology. “Patients with elevated preoperative CA19-9 that normalizes after curative resection are associated with comparable long-term survival outcomes compared to those with normal preoperative CA19-9, whereas…
China’s NMPA Accepts ALGS New Drug Application for Maralixibat
China’s National Medical Products Administration (NMPA) accepted the New Drug Application (NDA) for CAN108 (maralixibat) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) aged 1 year and above. “That the NMPA has accepted the NDA, which we anticipate will also receive priority review, for CAN108 for [ALGS], our first candidate in…
Conditional Survival Rates Vary Following Intrahepatic Cholangiocarcinoma Surgery
Conditional survival rates vary across stage categories in patients with nonmetastatic intrahepatic cholangiocarcinoma following surgery, according to a new study published in the Scandinavian Journal of Gastroenterology. This finding is important as it has implications in individualized clinical counseling and decision‐making for the disease. To examine the conditional survival rates of patients with nonmetastatic intrahepatic…
Cost-Effectiveness of LAL-D Orphan Drugs Remains Controversial
Orphan drugs used to treat diseases caused by inborn errors of metabolism like lysosomal acid lipase deficiency (LAL-D) show many disparities in effectiveness and a high economic impact, according to a new study published in Anales de Pediatría. However, they have an easy toxicity profile to manage. “The cost-effectiveness ratio for orphan drugs is a…
New Assay Can Measure Lower Active AAT Levels in AATD
A new method of measuring the levels of alpha-1 antitrypsin (AAT) activity in a sensitive and specific manner was discovered, as published in the Journal of Pharmaceutical and Biomedical Analysis. “The data obtained demonstrated the ECFISA [elastase complex formation immunosorbent assay] as an accurate, precise, selective, and very sensitive method for AAT activity measurement at…
- Loading...
Loading...
Next post in AATD News Briefs
Loading... 
Loading...