Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.

Background: In X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function of the ALD protein. Cerebral adrenoleukodystrophy is characterized by demyelination and neurodegeneration. Disease progression, which leads to loss of neurologic function and death, can be halted only with allogeneic hematopoietic stem-cell transplantation.

Posterior reversible encephalopathy syndrome in cancer patients: a single institution retrospective study.

Posterior reversible encephalopathy syndrome (PRES) is a clinico-radiologic entity. Its management and outcome in the oncology population is limited because it is still difficult to identify despite an increasingly recognized occurrence. This is the largest retrospective study of PRES in cancer patients reported from a single institution.

Hemichorea in a patient with diabetic ketoacidosis.

Background: Chorea is a common presenting feature of metabolic disorders, including nonketotic hyperglycemia in patients with type 2 diabetes mellitus, but rarely has been reported in diabetic ketoacidosis, hypothyroidism and vitamin B12 deficiency.

Methods: Review the literature for reported cases of chorea as a presenting manifestation in metabolic disorders.

Results: We report a case of hemichorea in a patient with type 2 diabetes mellitus complicated by diabetic ketoacidosis.

Hematopoietic stem cell transplantation in the leukodystrophies: a systematic review of the literature.

Objective: The objective of this study is to systematically review the literature on worldwide numbers of leukodystrophy patients undergoing hematopoietic stem cell transplantation (HSCT) as well as the safety and efficacy of the procedure in this patient population.

Materials And Methods: A PubMed and EMBASE search up to June 2012 was conducted with a manual search of references from relevant articles. Selected studies were evaluated using internationally accepted criteria.