Subcutaneous tissue disorder

This phase 2 trial tests whether saracatinib, an oral drug originally developed for cancer, can prevent new abnormal bone growth in adults with fibrodysplasia ossificans progressiva (FOP), a rare genetic condition where muscle and soft tissue turn into bone. Twenty adults with ac…

This study tests an experimental drug, garetosmab, in 63 adults with fibrodysplasia ossificans progressiva (FOP), a rare condition where soft tissues turn into bone. The goal is to see if the drug safely reduces new bone growth and painful flare-ups. Participants receive either t…

This study tests whether a device called Sonablate can safely use high-intensity focused ultrasound (HIFU) to treat vein problems like varicose veins, leg ulcers, and certain birth defects. About 30 adults with these conditions will receive the treatment, which does not involve c…

This program offers garetosmab to adults with fibrodysplasia ossificans progressiva (FOP) who finished the main OPTIMA study. The goal is to provide the drug before it is officially approved. Participants must meet specific health criteria to join.

This study tests an experimental drug called andecaliximab in 92 children and adults with fibrodysplasia ossificans progressiva (FOP), a rare genetic condition where soft tissues turn into bone. The goal is to see if the drug can safely reduce new bone growth and flare-ups. Parti…

ScreenPlus is a large pilot program that offers families the option to have their newborn screened for a panel of rare genetic disorders, in addition to standard newborn screening. The study aims to screen 100,000 infants born at eight hospitals in New York. Researchers will eval…

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This study uses a special camera and a safe dye to take pictures of the lymphatic system, which helps move fluid in the body. Researchers want to see how this system works in healthy people and in those with conditions like lymphedema or lipedema. The goal is to find patterns tha…

This phase 2 trial tests whether CBL-514 injections can reduce pain from lipomas in people with Dercum's disease. Nineteen adults with at least 4 painful lipomas will receive either the drug or a placebo. The main goal is to see if the treatment lowers pain scores.

This study tests whether 12 weeks of high-intensity interval training (HIIT) can reduce pain and improve quality of life in 62 women with lipedema, a chronic fat disorder causing painful leg swelling. Participants will do HIIT three times a week and then switch to usual care, or …

This national study in Norway is testing whether liposuction can reduce pain and improve quality of life for women with lipedema, a chronic condition causing painful fat buildup in the limbs. Researchers will compare early versus delayed liposuction, along with standard treatment…