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Showing 1 to 4 of 4 for “"Adrenoleukodystrophy"”.

  1. Mitigating oxidative stress in childhood cerebral adrenoleukodystrophy -an investigation of N-acetylcysteine pharmacology

    Adrenoleukodystrophy (ALD) is an X-linked genetic disorder which affects the adrenal glands, peripheral neuronal system, the spinal cord and white matter of central nervous system (CNS). It is a progressive neurology disorder with incidence of 1 in 17,000 newborns. ALD is caused by mutations in the …

    umn Repository record for Mitigating oxidative stress in childhood cerebral adrenoleukodystrophy -an investigation of N-acetylcysteine pharmacology (opens in a new tab)

  2. Chimerizmo analizė atskirose ląstelių populiacijose po alogeninės kraujodaros kamieninių ląstelių transplantacijos vaikams /

    … leukemia (ALL), Fanconi anemia (FA) and adrenoleukodystrophy (ALD) were included into the study. Thereafter the doctoral thesis was accomplished at the Vilnius University where evaluation of the own experience of chimerism analysis in pediatric patients was performed. Chimerism was …

    vilnius Repository record for Chimerizmo analizė atskirose ląstelių populiacijose po alogeninės kraujodaros kamieninių ląstelių transplantacijos vaikams / (opens in a new tab)

  3. Magnetic resonance spectroscopic imaging using parallel transmission at 7T

    … in studies of Late-Onset Tay- Sachs (LOTS), adrenoleukodystrophy (ALD) and brain tumors. A major contribution of this thesis is pTx excitation design for CSI to provide spectral-spatial mitigation of the B1+ inhomogeneities at 7T. Novel pTx RF designs are proposed and demonstrated to yield …

    mit Repository record for Magnetic resonance spectroscopic imaging using parallel transmission at 7T (opens in a new tab)

  4. Bayesian Hierarchical Models for Data Extrapolation and Analysis in Rare and Pediatric Disease Clinical Trials

    … turns to methods useful in the study of X-linked adrenoleukodystrophy (X-ALD), a rare neurodegenerative disease for which Lorenzo’s Oil (LO) is one of the few available treatments. We offer a hierarchical Bayesian statistical approach to understanding the pharmacokinetics (PK) and pharmacodynamics …

    umn Repository record for Bayesian Hierarchical Models for Data Extrapolation and Analysis in Rare and Pediatric Disease Clinical Trials (opens in a new tab)