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About 665 results

ALLMedicine™ Adrenoleukodystrophy Center

Research & Reviews  254 results

A Longitudinal Analysis of Early Lesion Growth in Presymptomatic Patients with Cerebral...
https://doi.org/10.3174/ajnr.A7250
AJNR. American Journal of Neuroradiology; Mallack EJ, Askin G et. al.

Sep 11th, 2021 - Cerebral adrenoleukodystrophy is a devastating neurological disorder caused by mutations in the ABCD1 gene. Our aim was to model and compare the growth of early cerebral lesions from longitudinal MRIs obtained in presymptomatic patients with progr...

Differential outcomes for frontal versus posterior demyelination in childhood cerebral ...
https://doi.org/10.1002/jimd.12435
Journal of Inherited Metabolic Disease; Gupta AO, Nascene DR et. al.

Sep 10th, 2021 - In the most common variant of childhood cerebral adrenoleukodystrophy (cALD), demyelinating brain lesions are distributed predominately in parieto-occipital white matter. Less frequently, lesions first develop in frontal white matter. This matched...

Magnetic resonance spectroscopy as marker for neurodegeneration in X-linked adrenoleuko...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8405970
NeuroImage. Clinical; van de Stadt SIW, Schrantee A et. al.

Aug 31st, 2021 - X-linked adrenoleukodsytrophy (ALD) is a genetic neuro-metabolic disorder, causing a slowly progressive myelopathy in adult male and female patients. New disease modifying therapies for myelopathy are under development. This calls for new (imaging...

Diverse clinical manifestations of X-linked adrenoleukodystrophy in a Chinese family wi...
https://doi.org/10.1097/YPG.0000000000000292
Psychiatric Genetics; Zhang L, Zhao SL et. al.

Aug 5th, 2021 - This study summarized the clinical characteristics of X-linked adrenoleukodystrophy (X-ALD) patients in this family, and two different manifestations of the same variants in a Chinese family were reported in this article. That conducted a follow-u...

Population Pharmacokinetic Analysis of N-Acetylcysteine in Pediatric Patients With Inhe...
https://doi.org/10.1002/jcph.1943
Journal of Clinical Pharmacology; Sahasrabudhe SA, Kartha RV et. al.

Jul 19th, 2021 - N-acetylcysteine (NAC) has been used in patients with cerebral adrenoleukodystrophy as an antioxidant agent in association with hematopoietic stem cell transplant (HSCT). However, an understanding of the pharmacokinetic characteristics of intraven...

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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 6th, 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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News  10 results

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

Mar 22nd, 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...

Neurocognitive Decline and Motor Regression in a 34-Year-Old Woman
https://reference.medscape.com/viewarticle/845434_3

Apr 23rd, 2018 - Discussion This patient's radiologic data and clinical picture were consistent with metachromatic leukodystrophy (MLD). Because the patient had already progressed to a bedridden, quadriparetic state, she was referred for rehabilitation, and the fa...

Bluebird to Seek Approvals for Three Treatments by End of 2019
https://www.medscape.com/viewarticle/891095

Jan 10th, 2018 - (Reuters) - U.S. biotech bluebird bio Inc, which does not yet have any marketed products, expects to be able to file for regulatory approval of three novel treatments by the end of 2019, Chief Executive Nick Leschly announced on Tuesday. Bluebird ...

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 12th, 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 ...

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Patient Education  1 results see all →