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About 1,475 results

ALLMedicine™ Adrenoleukodystrophy Center

Research & Reviews  713 results

False-positive very long-chain fatty acids in a case of autoimmune adrenal insufficiency.
https://doi.org/10.1515/jpem-2020-0652
Journal of Pediatric Endocrinology & Metabolism : JPEM; Zhu J, Breault DT

Apr 5th, 2021 - X-linked adrenoleukodystrophy (ALD) affects up to 25% of boys diagnosed with adrenal insufficiency in childhood. Because early identification of these individuals can be lifesaving, all boys with new-onset primary adrenal insufficiency should be t...

Long-term follow-up of Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodys...
https://doi.org/10.1089/hum.2021.053
Human Gene Therapy; Bougneres P, Hacein-Bey-Abina S et. al.

Apr 1st, 2021 - BACKGROUND
In 2009, cerebral adrenoleukodystrophy (c-ALD) became the first brain disease to be treated with lentiviral-based hematopoietic stem cell gene therapy with the ABCD1 gene in four boys (P1-P4) who had demyelinating lesions expected to be...

Dentate Nucleus Signal Intensity Changes in Children with Adrenoleukodystrophy in Compa...
https://doi.org/10.1111/jon.12844
Journal of Neuroimaging : Official Journal of the America... Ozturk K, Nascene D

Mar 30th, 2021 - To determine whether cerebral adrenoleukodystrophy (cALD) or brain irradiation in patients with primary brain tumor affects T1-weighted imaging (T1WI) signal intensity (SI) of the dentate nucleus (DN) in a pediatric cohort who had received consecu...

Allogeneic hematopoietic stem cell transplantation in patients with childhood cerebral ...
https://doi.org/10.1111/petr.14015
Pediatric Transplantation; Yalcin K, Çelen SS et. al.

Mar 29th, 2021 - ALD is a rare X-linked peroxisomal metabolic disorder with many distinct phenotypes of disease that emerge on a wide scale from adrenal insufficiency to fatal cALD which progresses to a vegetative state within a few years. Currently, HSCT is the o...

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Guidelines  1 results

Adrenoleukodystrophy: Guidance for Adrenal Surveillance in Males Identified by Newborn ...
https://doi.org/10.1210/jc.2018-00920
The Journal of Clinical Endocrinology and Metabolism; Regelmann MO, Kamboj MK et. al.

Oct 5th, 2018 - Adrenoleukodystrophy (ALD) is a peroxisomal disorder associated with neurologic decompensation and adrenal insufficiency. Newborn screening for ALD has recently been implemented in five states with plans to expand to all 50 states in the United St...

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Clinicaltrials.gov  739 results

False-positive very long-chain fatty acids in a case of autoimmune adrenal insufficiency.
https://doi.org/10.1515/jpem-2020-0652
Journal of Pediatric Endocrinology & Metabolism : JPEM; Zhu J, Breault DT

Apr 5th, 2021 - X-linked adrenoleukodystrophy (ALD) affects up to 25% of boys diagnosed with adrenal insufficiency in childhood. Because early identification of these individuals can be lifesaving, all boys with new-onset primary adrenal insufficiency should be t...

Long-term follow-up of Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodys...
https://doi.org/10.1089/hum.2021.053
Human Gene Therapy; Bougneres P, Hacein-Bey-Abina S et. al.

Apr 1st, 2021 - BACKGROUND
In 2009, cerebral adrenoleukodystrophy (c-ALD) became the first brain disease to be treated with lentiviral-based hematopoietic stem cell gene therapy with the ABCD1 gene in four boys (P1-P4) who had demyelinating lesions expected to be...

Dentate Nucleus Signal Intensity Changes in Children with Adrenoleukodystrophy in Compa...
https://doi.org/10.1111/jon.12844
Journal of Neuroimaging : Official Journal of the America... Ozturk K, Nascene D

Mar 30th, 2021 - To determine whether cerebral adrenoleukodystrophy (cALD) or brain irradiation in patients with primary brain tumor affects T1-weighted imaging (T1WI) signal intensity (SI) of the dentate nucleus (DN) in a pediatric cohort who had received consecu...

Allogeneic hematopoietic stem cell transplantation in patients with childhood cerebral ...
https://doi.org/10.1111/petr.14015
Pediatric Transplantation; Yalcin K, Çelen SS et. al.

Mar 29th, 2021 - ALD is a rare X-linked peroxisomal metabolic disorder with many distinct phenotypes of disease that emerge on a wide scale from adrenal insufficiency to fatal cALD which progresses to a vegetative state within a few years. Currently, HSCT is the o...

see more →

News  13 results

Gene Therapy for ALD Prevents Major Functional Disabilities
https://www.medscape.com/viewarticle/947868

Mar 21st, 2021 - An investigational gene therapy, elivaldogene autotemcel (eli-cel, Bluebird Bio), appears to hold at bay the devastating neurodegenerative disease cerebral adrenoleukodystrophy (ALD).   Two years after receiving a single gene therapy treatment, th...

Gene Therapy May Benefit Patients With Cerebral ALD
https://www.mdedge.com/neurology/article/153999/rare-diseases/gene-therapy-may-benefit-patients-cerebral-ald

Dec 11th, 2017 - KANSAS CITY, MO—Lentiviral gene therapy halts inflammation and demyelination in patients with cerebral adrenoleukodystrophy (ALD), according to research presented at the 46th Annual Meeting of the Child Neurology Society and published in the New E.

Treatment Success in Fatal Disorder in Children
https://www.medscape.com/viewarticle/886974

Oct 11th, 2017 - A gene therapy for a fatal neurologic degenerative condition affecting children has shown major success in a clinical trial. In the STARBEAM trial, adrenoleukodystrophy  stabilized in 15 of 17 children who received the gene therapy, with no major ...

Gene therapy for cerebral adrenoleukodystrophy shows promise
https://www.mdedge.com/neurology/article/149021/pediatrics/gene-therapy-cerebral-adrenoleukodystrophy-shows-promise
Bianca Nogrady

Oct 9th, 2017 - Gene therapy using autologous hematopoietic stem cells appears safe and effective as a treatment for early-stage cerebral adrenoleukodystrophy, according to an interim analysis of results from the STARBEAM study. X-linked adrenoleukodystrophy is c.

Small Gene Therapy Trial Success for Rare Degenerative Brain Disease
https://www.medpagetoday.com/neurology/generalneurology/68321

Oct 4th, 2017 - Action Points Note that this phase II/III trial of autologous stem cell transplant with gene therapy in boys with adrenoleukodystrophy showed promising efficacy results. Be aware that the new treatment was not compared directly to the standard of ...

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Patient Education  9 results see all →