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Publication

Potential Risks to Stable Long-term Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Children With Cerebral X-linked Adrenoleukodystrophy.

Jörn-Sven Kühl | Jana Kupper | Hermann Baqué | Wolfram Ebell | Jutta Gärtner | Christoph Korenke | Birgit Spors | Ingo G Steffen | Gabriele Strauss | Sebastian Voigt | Bernhard Weschke | Almuth Weddige | Wolfgang Köhler | Robert Steinfeld
JAMA network open | 2018

Allogeneic hematopoietic stem cell transplantation is the standard intervention for childhood cerebral X-linked adrenoleukodystrophy. However, the pretransplant conditions, demyelination patterns, complications, and neurological outcomes of this therapy are not well characterized.

Pubmed ID: 30646031

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