Niemann-Pick disease type B

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 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 phase 2 trial tested the drug samotolisib in 18 children and young adults with advanced solid tumors, non-Hodgkin lymphoma, or histiocytic disorders that had spread, returned, or stopped responding to treatment. Only patients whose tumors had specific mutations in the TSC or…

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 study tested whether adding rituximab (a drug that calms certain immune cells) to standard mycophenolate mofetil treatment helps people with interstitial lung disease whose lungs are not responding to initial therapy. 122 adults with various ILD types received either rituxim…

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 is testing a portable, non-invasive breath analyzer that measures tiny particles in exhaled breath to screen for conditions like type 2 diabetes, liver disease, kidney disease, and lung disease. Researchers will enroll healthy volunteers and people with known diagnoses…

This study tested whether a diode laser can act as a biomarker to measure neuropathic pain in people with peripheral neuropathy. Researchers compared pain responses to a lidocaine patch versus a placebo patch in 75 participants. The goal was to see if the laser test could disting…

This study looked at three ways to take a small sample of lung tissue (biopsy) in 60 adults with interstitial lung diseases. The goal was to find which method gives the most accurate diagnosis and causes the fewest side effects. The methods tested were forceps biopsy, cryobiopsy …

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 tested a new MRI technique that can take pictures of children's lungs without using radiation or injecting contrast dye. 72 children aged 6-16 had both a standard CT scan and this new MRI. The goal was to see if the MRI could produce clear images of the lungs, potentia…

This trial investigates whether adding high-flow nasal oxygen during exercise training can improve endurance and quality of life for people with chronic fibrotic interstitial lung disease (ILD). Participants undergo a 4-week pulmonary rehabilitation program, receiving either high…

This study tested whether giving a small amount of s-ketamine during chest surgery could reduce the risk of postoperative delirium (sudden confusion) in patients aged 60 and older. Over 500 participants were randomly assigned to receive either s-ketamine or another drug (dexmedet…

This study tested freezing the nerves between the ribs during robot-assisted lung surgery to see if it reduces pain and the need for painkillers afterward. 33 adults having lung surgery were randomly assigned to get the nerve freeze or standard pain medicine. Researchers tracked …

This study looked at whether giving a low dose of a muscle relaxant (rocuronium) during bronchoscopy—a procedure where a tube is inserted into the lungs—could reduce complications like throat spasms and coughing. 153 adults having elective bronchoscopy were split into two groups:…

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 follows 1500 adults with interstitial lung disease to understand how the condition changes over time. Researchers will track symptoms, lung function, and treatments used. The goal is to build a detailed picture of the disease's course and improve future care.

This study looked at immune cells called alveolar macrophages in the lungs of 18 people with non-infectious lung diseases. Researchers used a technique called flow cytometry to study the surface proteins on these cells. The goal was to better understand the role of these cells in…

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 looked at ways to screen for pulmonary hypertension (high blood pressure in the lungs) in people who already have interstitial lung disease. The goal was to find this complication earlier. 314 adults with various types of lung disease took part in the screening tests.

This study looked at whether certain genes make people who are regularly around pigeons more likely to develop a type of lung disease called hypersensitivity pneumonitis. Researchers analyzed the DNA of 475 pigeon fanciers to find genetic markers linked to lung scarring. The goal…

This study looked at 86 ICU patients with a lung condition called interstitial syndrome. Researchers compared a lung ultrasound score with a standard blood oxygen test to see if the ultrasound could reliably measure how severe the condition was. They repeated both tests at the st…

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 93 people with lung disease who get short of breath when active. Participants did two 6-minute walk tests: one at their fastest pace and one at their normal walking speed. The goal was to see if the normal-speed test gives a more accurate picture of when they…

This study tested a special MRI technique using a harmless gas to see how well it can show changes in the lungs of people with interstitial lung disease. Researchers worked with 46 volunteers to find the best way to use this scan. The goal is to create a standard method that doct…

This study tested a special MRI technique called Ultra Short Echo Time (UTE) to see if it can take clearer pictures of the lungs than traditional MRI. Researchers collected images and health information from people with and without lung disease to help doctors better understand l…

This study tested whether having a dietitian help patients with lung disease order meals during a hospital stay could improve their nutrition and reduce the chance of being readmitted after discharge. 41 patients at nutritional risk took part. Researchers measured daily food inta…

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…