DYSKERATOSIS CONGENITA

This study tests a stem cell transplant method for people with serious immune disorders or bone marrow failure. The goal is to see if a gentler pre-transplant treatment, followed by a drug to prevent rejection, can help donor cells take over and restore healthy blood and immune f…

This study tests a special stem cell transplant that removes certain immune cells to lower the risk of graft-versus-host disease (GVHD) and reduce the need for long-term immune-suppressing drugs. It is for people with inherited bone marrow failure disorders like Fanconi anemia. T…

This expanded access program offers a special stem cell filtering technique (CD34+ selection) to patients with severe blood disorders like sickle cell disease and immune deficiencies. The goal is to lower the risk of graft-versus-host disease, a serious complication where donor c…

This early-stage trial tests whether a combination of two natural substances (deoxycytidine and deoxythymidine) is safe for people with telomere biology disorders—rare conditions that cause premature aging, bone marrow failure, and lung disease. Up to 36 participants aged 1 to 70…

This research study aims to discover new genes that may cause cancer to run in families. Researchers will collect blood samples and health information from 1,500 people in families where multiple members have had childhood cancers. Participants will not receive direct treatment o…

This study looks at families with inherited bone marrow failure syndromes (like Fanconi anemia) to understand why they have a higher risk of cancer. Researchers will track up to 4,000 participants over time using questionnaires, medical records, and lab tests. The goal is to find…

This study collects blood, bone marrow, and other samples from people with non-cancerous blood diseases and their family members. Researchers will analyze DNA and other markers to find genetic causes and factors that affect disease severity and treatment response. The goal is to …