Osteosclerosis

This study tests a new gene therapy for children with a severe inherited bone disease called osteopetrosis. The therapy uses the child's own blood stem cells, modified to fix the genetic defect, and then returned to the body. The goal is to improve blood cell production and survi…

This study tracks 50 children and adults with non-malignant disorders like immune deficiencies and anemias who receive a stem cell transplant after a reduced-intensity chemotherapy regimen. The goal is to see if this approach improves survival and reduces severe graft-versus-host…

This study is testing a less intense chemotherapy and radiation regimen before a stem cell transplant for children and young adults up to age 55 with non-cancerous blood disorders like immune deficiencies, anemias, and metabolic diseases. The goal is to see if this gentler prepar…

This study compares a newer painkiller called oliceridine with two standard drugs, morphine and sufentanil, for pain relief after lumbar spine surgery. Ninety adults will receive one of the three drugs through a patient-controlled pump. The main goal is to see how well each drug …

This study tests whether virtual reality exercise games can improve balance, reduce fall risk, and boost quality of life in postmenopausal women aged 50–75 with osteoporosis. Fifty participants will either do VR exercises or standard care. The goal is to see if fun, interactive g…

This study follows adults with melorheostosis, a rare disease that causes bone thickening, pain, and joint problems. Researchers will track how the disease progresses over time, collect genetic samples from patients and their relatives, and look for biomarkers. The goal is to bet…

This study aims to find the genes and genetic changes that cause craniometaphyseal dysplasia (CMD), a rare disorder that leads to abnormal bone growth in the skull and other bones. Researchers will analyze blood and tissue samples from 600 people, including affected individuals a…

This study follows 1500 people with rare genetic brain disorders to learn how these diseases progress. Researchers measure thinking, movement, and daily living skills over time, and also look at brain scans and body fluids. The goal is to better understand the diseases and how tr…