Indications for pediatric hematopoietic stem cell transplantation: consensus presented at the First Meeting on Brazilian Hematopoietic Stem Cell Transplantation Guidelines - Brazilian Society of Bone Marrow Transplantation, Rio de Janeiro, 2009 [PDF]
, 2010 The Brazilian Bone Marrow Transplant Society (SBTMO) held its First Meeting on Bone Marrow Transplant Guidelines in 2009. A working group of hematologists and oncologists with experience in pediatrics was formed to review evidence-based indications for ...
Bonfim, Carmem Maria S. +6 more
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Bone Marrow Transplant in Adrenoleukodystrophy
Pediatric Neurology Briefs, 1997 Three children with adrenoleukodystrophy (ALD) were treated with allogeneic bone marrow transplantation (BMT) at Huddinge University Hospital, Sweden.
J Gordon Millichap
doaj +1 more source
Adeno-associated virus and lentivirus vectors: a refined toolkit for the central nervous system. [PDF]
, 2016 The last two decades have witnessed the increasing instrumentalization of viruses, which have progressively evolved into highly potent gene transfer vehicles for a wide spectrum of applications.
Blessing, D., Déglon, N.
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Cell Proliferation, Volume 59, Issue 3, March 2026.Pioglitazone suppresses mitochondrial excessive ROS generation and COX‐2 level through inhibition of Drp‐1 mediated mitochondrial fission in an AMPK‐dependent manner in LS. ABSTRACT Loss of function mutations of NDUFS4 resulted in Leigh syndrome, which is a progressive neurodegenerative disease and characterized by mitochondrial oxidative stress ...
Jie Luo +5 more
wiley +1 more source
Lorenzo Oil Therapy for Adrenoleukodystrophy
Pediatric Neurology Briefs, 1993 Dietary therapy with glycerol trioleate and glycerol trierucate (Lorenzo oil) was tested in 108 adult patients with adrenomyeloneuropathy phenotype of adrenoleukodystrophy (ALD) at Johns Hopkins Hospital and the Kennedy Krieger Institute, Baltimore, MD.
J Gordon Millichap
doaj +1 more source
Genetic analysis and prenatal diagnosis of 76 Chinese families with X‐linked adrenoleukodystrophy
Molecular Genetics & Genomic Medicine, 2022 Background Variants in the ATP binding cassette protein subfamily D member 1 (ABCD1) gene are known to cause X‐linked adrenoleukodystrophy (X‐ALD). This study focused on the characteristics of ABCD1 variants in Chinese X‐ALD families and elucidated the ...
Siwen Liu +8 more
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Magnetic resonance imaging (MRI) findings in white matter disease of brain [PDF]
, 2008 Demyelinating and dysmyelinating white matter diseases are important components of neurological problems. Recently, Magnetic Resonance Imaging (MRI) has played a key role in diagnoses of white matter diseases.
Ahsan, Humera +5 more
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Making Advanced Therapies Affordable and Accessible: Two Strategic Approaches
Developing World Bioethics, Volume 26, Issue 1, Page 66-78, March 2026.ABSTRACT This article explores two complementary strategies for addressing the affordability and access challenges facing advanced therapies. As high development costs and limited market access have led to the withdrawal of several therapies, the article examines how these barriers create ‘valleys of death’ that prevent innovation from reaching ...
Ubaka Ogbogu, Lauren Albrecht
wiley +1 more source
Virchow-Robin spaces : an anatomic variant or a pathologic sign? [PDF]
, 2009 Virchow-Robin spaces surround blood vessels. Their walls are formed by prolongations of the pia mater and they have no communication with the subarachnoid space. VRS are often seen as well-delineated foci of cerebrospinal fluid signal on MR images.
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