ALLMedicine™ Adrenomyeloneuropathy Center
Research & Reviews 50 results
https://doi.org/10.1002/jimd.12457
Journal of Inherited Metabolic Disease; Keller JL, Eloyan A et. al.
Nov 20th, 2021 - Current outcomes used to evaluate adrenomyeloneuropathy are limited by rater bias, not sensitive to preclinical changes, and require years to decades to detect disease progression. Quantitative outcomes are needed that detect meaningful change in ...
https://doi.org/10.1016/j.parkreldis.2021.10.006
Parkinsonism & Related Disorders; Hsu SL, Chen YH et. al.
Oct 15th, 2021 - Adrenoleukodystrophy (ALD) is an X-linked peroxisomal disorder caused by mutations in the ABCD1 gene. The clinical manifestations of ALD vary widely with some patients presenting with adrenomyeloneuropathy (AMN) that resembles the phenotype of her...
https://clinicaltrials.gov/ct2/show/NCT05008874
Aug 17th, 2021 - Progressive weakness and spasticity of the legs are characteristics of numerous disorders and conditions, including those that are inherited neurological disorders. Adrenomyeloneuropathy (AMN) is an example of an inherited form of spastic parapleg...
https://doi.org/10.1002/ana.26141
Annals of Neurology; Schäfer L, Roicke H et. al.
Jun 10th, 2021 - X-linked adrenoleukodystrophy (X-ALD) is a rare genetic disorder characterized by progressive demyelination ranging from mild myelopathic forms (adrenomyeloneuropathy [AMN]) to severe cerebral variants (adult cerebral adrenoleukodystrophy [ACALD])...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7985512
Nature Communications; Weinhofer I, Rommer P et. al.
Mar 24th, 2021 - X-linked adrenoleukodystrophy (X-ALD), the most frequent monogenetic disorder of brain white matter, is highly variable, ranging from slowly progressive adrenomyeloneuropathy (AMN) to life-threatening inflammatory brain demyelination (CALD). In th...
Clinicaltrials.gov 2 results
https://clinicaltrials.gov/ct2/show/NCT05008874
Aug 17th, 2021 - Progressive weakness and spasticity of the legs are characteristics of numerous disorders and conditions, including those that are inherited neurological disorders. Adrenomyeloneuropathy (AMN) is an example of an inherited form of spastic parapleg...
https://clinicaltrials.gov/ct2/show/NCT04687007
Dec 30th, 2020 - X-linked Adrenoleukodystrophy (X-ALD) is one of the most frequent inborn error of metabolism caused by mutations in the ABCD1 gene, which codes for the transporter of saturated very long-chain fatty acids (VLCFA) for peroxisomal degradation, thus ...
News 1 results
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.
