Bardet-Biedl syndrome study in a Syrian teen

This trial tests a drug called setmelanotide for people with Bardet-Biedl Syndrome (a rare genetic condition) or other severe genetic obesity. It looks at how well the drug works in real-world settings, not just in a lab.

This study is a registry that collects health and genetic information from people with Bardet-Biedl syndrome. It may help researchers better understand the condition and improve future care by building a clear group of eligible patients.

This study follows people with Bardet-Biedl syndrome (BBS) or Alström syndrome (ALMS) to spot early vision, metabolic (body chemistry/energy), and kidney problems. You may be asked to have clinical checks over time to help researchers better understand how these complications develop.

This early-phase trial tests a gene therapy called AXV-101 for children with vision loss caused by BBS1 genetic mutations. The goal is to see if it can help slow or stop retinal damage.

This study looks at the health records and blood tests of children and young adults with Behçet disease to learn more about the condition. It also compares their results with healthy children to find clues that may help doctors better understand and treat Behçet disease.

This study follows people with Baker Gordon syndrome over time to better understand the condition. Participants and their caregivers share medical records and complete tests and questionnaires.