Telomere syndrome

This study tested whether low doses of the hormone drug danazol can slow the shortening of telomeres—the protective caps on DNA—in people with telomere diseases. These conditions can cause bone marrow failure, lung scarring, and liver damage. Eighteen participants took danazol fo…

This study tested a drug called seladelpar in 96 adults with primary biliary cholangitis (PBC), a rare liver disease. Participants had not responded well or could not tolerate the standard treatment. The goal was to see if seladelpar could lower a key liver enzyme (alkaline phosp…

This study tested a drug called saroglitazar magnesium in 196 adults with primary biliary cholangitis (PBC), a chronic liver disease. Participants either took the drug or a placebo to see if it improved liver function markers. The goal was to lower certain liver enzymes and bilir…

This study tested a new drug called CNP-104 in 42 adults with primary biliary cholangitis, a rare liver disease, who did not get better with standard treatments. The drug is designed to calm the immune system's attack on the bile ducts. The main goals were to check safety and see…

This early-phase study tested whether adding fenofibrate to standard treatment (ursodeoxycholic acid) helps people newly diagnosed with primary biliary cholangitis, a chronic liver disease. Thirty adults participated to see if the combination could normalize liver enzyme levels a…

This first-in-human study tested a single dose of PVT201, a nanoparticle-based drug, in 24 people: healthy volunteers and patients with primary biliary cholangitis (PBC) or primary sclerosing cholangitis (PSC). The main goal was to check safety and how the drug moves through the …

This study tested whether adding bezafibrate to the standard drug obeticholic acid helps people with primary biliary cholangitis, a rare liver disease. 72 adults took part, and researchers measured changes in a key liver enzyme over 12 weeks. The goal is to see if the combination…

This study tested how well a special MRI scan called LiverMultiScan can measure liver fat, iron, and scarring. 61 adults with various liver diseases had up to 6 scans on different machines. The goal was to see if the results are consistent when repeated on the same scanner or on …

This study tested a 10-week online wellness program for women with primary biliary cholangitis (PBC), a chronic liver disease that causes fatigue, itching, brain fog, and emotional distress. The program included guided breathing, meditation, gentle movement, and optional nutritio…

This study tested a 12-week online mind-body program for adults with chronic conditions like heart failure, cancer, and liver disease. The program included mindful movement, meditation, breathwork, and coping skills. Researchers measured changes in anxiety, depression, fatigue, a…

This completed Phase 1 study looked at how the drug seladelpar (Livdelzi) is processed in 24 people with primary biliary cholangitis (PBC) who also have liver impairment. Researchers measured drug levels in the blood and urine to see how the liver condition affects the drug. The …

This study looked at whether ultrasound and stiffness measurements of the liver and spleen can predict a serious condition called portal hypertension in people with common variable immunodeficiency (CVID). Researchers examined 250 CVID patients during routine visits, using non-in…

This study tested a wearable sensor called ADAM to measure scratching and sleep patterns in 30 people with primary biliary cholangitis (PBC), a rare liver disease that often causes intense itching and poor sleep. Participants wore the sensor for 7 nights across four 7-day periods…

This study followed 711 adults with primary biliary cholangitis (PBC) for 5 years to see how the disease is managed in everyday medical practice. Researchers collected data on treatments, lab results, and side effects, and also stored blood samples for future genetic research. Th…

This study looked at 27 adults with a below-knee amputation who use a prosthesis. Researchers measured how satisfied they were with their prosthetic, how they felt about their body image, and how well they could do daily activities and participate in social life. The goal was to …

This study looks at the genetic makeup of 1,500 people with Primary Biliary Cirrhosis (PBC), a rare liver disease, along with their close relatives. By comparing genes, researchers hope to find inherited factors that make someone more likely to develop PBC. The goal is to better …