Results 91 to 100 of about 18,427 (209)
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
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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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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
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Molecular Genetics &Genomic Medicine, Volume 14, Issue 4, April 2026.We developed and implemented a 500‐gene panel for phenotype‐driven genetic testing of Mendelian disorders in South Africa's public healthcare system, achieving a 46% diagnostic yield. This platform supports scalable, cost‐effective rare disease diagnosis and lays the foundation for broader genetic services in resource‐limited settings.
Nadia Carstens +3 more
wiley +1 more source
A Review of Genetic Transfer Therapy Trials from a Regulatory Perspective for the Scientific Understanding [PDF]
, 2013 The present work reviews the safety and efficacy concerns of the Recombinant DNA Advisory Committee (RAC) regarding Gene Transfer Therapy (GTT) clinical trials.
Olguin, Arturo
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European Journal of Haematology, Volume 116, Issue 4, Page 336-349, April 2026.ABSTRACT Allogeneic hematopoietic stem cell transplantation (HSCT) is increasingly used to treat malignant and non‐malignant diseases. Following allogeneic HSCT, patients are particularly vulnerable to vaccine‐preventable diseases (VPD) because conditioning depletes immune cells, including memory cells.
Hélène Buvelot +3 more
wiley +1 more source
HIV-1 infection of microglial cells in a reconstituted humanized mouse model and identification of compounds that selectively reverse HIV latency. [PDF]
, 2017 Most studies of HIV latency focus on the peripheral population of resting memory T cells, but the brain also contains a distinct reservoir of HIV-infected cells in microglia, perivascular macrophages, and astrocytes.
Alvarez-Carbonell, David +4 more
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ABCD1 Gene Mutations: Mechanisms and Management of Adrenomyeloneuropathy
The Application of Clinical Genetics, 2022 Alyssa M Volmrich,1 Lauren M Cuénant,1 Irman Forghani,2 Sharon L Hsieh,3 Lauren T Shapiro1 1Department of Physical Medicine & Rehabilitation, University of Miami Miller School of Medicine, Miami, FL, USA; 2Department of Human Genetics, University of ...
Volmrich AM +4 more
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European Journal of Haematology, Volume 116, Issue 4, Page 443-449, April 2026.ABSTRACT This retrospective study evaluates the efficacy and safety of donor lymphocyte infusion (DLI) after allogeneic hematopoietic stem cell transplantation (HSCT) in children. We describe the long‐term use of preemptive, prophylactic, and therapeutic DLI with a gradual dose increase in half‐log increments.
Dinah Walther +7 more
wiley +1 more source
Indian Journal of Psychiatry, 2016 Adrenoleukodystrophy (ALD) is an X-linked disorder with diverse clinical presentations. A 30-year-old male, previously diagnosed with Addison's disease, on steroid supplementation for 18 years, presented to us with manic symptoms for 4 years.
K. S. Jyothi, Cyriac George, K. S. Shaji
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