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This study looked at whether the HPV vaccine works in girls aged 9 to 17 who are taking medicines that weaken their immune system, such as after an organ transplant or for diseases like lupus. The goal was to see if their bodies produce enough antibodies to protect against HPV. T…

This study tested a program to help veterans with heart disease or rheumatic conditions take their medications as prescribed. Over 5,000 participants received reminders via text or phone calls, with extra support if needed. The goal was to improve medication adherence and reduce …

This study looked at the long-term safety of a drug called ARO-APOC3 in adults with dyslipidemia (abnormal fat levels in the blood). Participants who completed a previous 12-month study could continue receiving the drug every 3 or 6 months. The goal was to see if the drug remains…

This study tested a low dose (10 mg) of the statin drug rosuvastatin in 242 Korean adults with high cholesterol or heart disease risk. The goal was to see how much it lowered 'bad' LDL cholesterol after 8 weeks. Results help guide cholesterol treatment in this population.

This study tested whether a drug called alirocumab, which lowers cholesterol, can reduce heart disease risk in people with HIV. The trial involved 118 adults aged 40 and older with well-controlled HIV and high heart disease risk. Participants received either alirocumab or a place…

This early-stage study tested a new medicine called ARO-ANG3 in 93 people, including healthy volunteers and patients with high cholesterol or high triglycerides. The main goal was to check if the drug is safe and how the body processes it. This research helps determine if the dru…

This study tested two different chemotherapy drugs (Treosulfan and Busulfan) given before a stem cell transplant in 106 children with serious non-cancer diseases like immune disorders, metabolic diseases, blood disorders, and bone marrow failure. The goal was to see which drug le…

This study looked at how well flu vaccines (seasonal and H1N1) work in 234 adults with autoimmune diseases like lupus or vasculitis, some of whom were on steroids or immunosuppressants. Researchers measured antibody levels three weeks after each shot to see if the vaccine provide…

This study tested a new method called global metabolomic profiling to diagnose inborn errors of metabolism, a group of rare genetic disorders. Researchers compared this approach to traditional testing in 240 participants. The goal was to see if the new method could more accuratel…

This study looked at how stretchy the skin and blood vessels are in people with Williams syndrome, a genetic condition that can cause heart problems. Researchers measured skin and pulse speed in 43 participants aged 5 to 70. The goal was to learn how these tissues change over tim…

This study created a registry of medical records from women with inborn errors of metabolism—rare conditions that affect how the body turns food into energy. Researchers collected data from past or current pregnancies and followed babies for one year after birth. The goal was to …

This study aimed to find new gene changes that cause rare lipid disorders. Researchers used advanced genetic testing to look at many genes at once in 140 people and their relatives. The goal was to discover causes that older methods missed.

This completed study looked at how the social networks of caregivers affect their stress and health when caring for someone with an inherited disease. Researchers surveyed over 680 participants, including family members and formal caregivers, to understand caregiving burden and s…

This study looked at 719 people, including patients with autoimmune connective tissue diseases like myositis, their blood relatives, and healthy volunteers. Researchers collected blood samples and medical histories to find genetic and environmental factors that may trigger these …

This completed study looked at how the COVID-19 pandemic and social distancing impacted the mental health of 259 adults with chronic inflammatory rheumatism (like rheumatoid arthritis or ankylosing spondylitis). Researchers used questionnaires to measure stress, depression, anxie…

This study looked at how fat-carrying particles called lipoproteins work in healthy people and those with high cholesterol or other lipid disorders. Researchers injected tiny, safe amounts of radioactive lipoproteins to track them in the body. The goal was to better understand ho…

This study tested a smartwatch and mobile app platform called Pedi@Ctivity to track physical activity in 362 adolescents, including those with chronic rheumatic disease. The goal was to collect big data and use AI to analyze activity patterns and create personalized exercise prog…

This study looked at over 13,000 people with chronic inflammatory rheumatism or autoimmune diseases who had or were suspected to have COVID-19. Researchers tracked how many developed severe COVID-19 requiring intensive care or leading to death. They also examined risk factors lik…

This study looked at 99 people with high heart risk to understand how a protein called CETP affects plaque buildup in arteries. Researchers measured CETP activity, artery thickness, and calcium scores. The goal was to learn more about heart disease, not to test a new treatment.

This study looked at over 2,400 people with rheumatic diseases who take methotrexate. Researchers compared those who had standard lab monitoring to those who had fewer blood tests. The goal was to see if reduced monitoring is as safe in terms of preventing organ damage or death.

This completed study looked at anxiety about COVID-19 in 200 adults with rheumatic diseases who were already taking either biologic or conventional disease-modifying drugs. Researchers called participants and asked them to fill out a questionnaire about their coronavirus-related …

This study looked at whether video or phone visits (tele-rheumatology) work as well as in-person visits for people with rheumatic diseases like arthritis. Over 650 adults took part, and researchers measured how satisfied they were with each type of visit and which they preferred.…

This study tested whether a powerful DNA test called high-throughput exome sequencing can find genetic causes of multiple birth defects in fetuses when standard exams fail. Researchers studied 100 fetuses with at least two malformations and no prior diagnosis. They compared the n…

This study tested whether a fast type of genetic test called rapid genome sequencing can find the cause of birth defects seen on ultrasound more quickly than current methods. Researchers included 184 pregnancies with certain ultrasound findings. The goal was to see if results cou…

This study measured two newer blood markers (calprotectin and serum amyloid protein) alongside standard tests in 20 children with juvenile arthritis and related conditions. The goal was to see if these markers could better track disease flare-ups in everyday clinic visits. The st…

This study looked at how many girls and young women aged 11 to 20 with chronic diseases (like diabetes or immune conditions) got the HPV vaccine, compared to those without chronic illness. Researchers reviewed records of 223 participants from a hospital in France. The goal was to…

This study collected blood, saliva, stool, urine, and skin samples from 19 people with unknown or poorly understood immune system problems. The goal was to learn how different parts of the immune system work together. No treatments or drugs were tested. Participants were followed…

This study tested a simple approach where doctors review pharmacy refill data during clinic visits and talk with patients about their medications. The goal was to see if this method is practical and acceptable for improving medication adherence in people with chronic rheumatic di…

This completed study looked at 32 people with known genetic changes in connective tissue genes to better understand how these changes affect overall health. Participants underwent a range of medical tests and exams over 3-5 days. The goal was to gather information, not to test a …