Early clinical course after hematopoietic stem cell transplantation in children with juvenile metachromatic leukodystrophy.
Judith BeschleMichaela DöringChristiane KehrerChrista RaabeUte BayhaManuel StrölinJudith BöhringerAndrea BevotNadja KaiserBenjamin BenderAlexander GrimmPeter LangIngo MüllerIngeborg Krägeloh-MannSamuel GroeschelPublished in: Molecular and cellular pediatrics (2020)
Our results show that if disease progression occurs, this happens early on after HSCT and proceeds faster than in non-transplanted children with juvenile MLD, indicating that HSCT may trigger disease progression.