New hope for rare genetic disorder: Lower-Dose chemo before transplant may cut risks
NCT ID NCT07316595
First seen Jan 07, 2026 · Last updated May 15, 2026 · Updated 17 times
Summary
This study tests a lower dose of the drug treosulfan before a stem cell transplant in 24 people with Nijmegen breakage syndrome, a rare genetic condition that weakens the immune system and raises cancer risk. The goal is to see if this approach can safely fix the immune problem and lower the chance of tumors, while avoiding severe side effects. Participants without cancer get a lower dose, and those with cancer get a slightly higher dose.
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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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