ALLMedicine™ Adrenomyeloneuropathy Center
Research & Reviews 66 results
https://clinicaltrials.gov/ct2/show/NCT04675749
Mar 8th, 2023 - X-linked adrenoleukodystrophy (X-ALD) is a hereditary white matter disorder caused by mutations in the ABCD1 gene leading to disturbances in the metabolism of fatty acids. This results in an accumulation of very long chain fatty acids (VLCFA) in t...
https://clinicaltrials.gov/ct2/show/NCT04687007
Mar 8th, 2023 - 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 ...
https://doi.org/10.1016/S1474-4422(22)00495-1
The Lancet. Neurology; Köhler W, Engelen M et. al.
Jan 22nd, 2023 - Adult patients with adrenoleukodystrophy have a poor prognosis owing to development of adrenomyeloneuropathy. Additionally, a large proportion of patients with adrenomyeloneuropathy develop life-threatening progressive cerebral adrenoleukodystroph...
https://clinicaltrials.gov/ct2/show/NCT05008874
Dec 13th, 2022 - 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/NCT04925349
Oct 12th, 2022 - X-linked Adrenoleukodystrophy (X-ALD), Metachromatic Leukodystrophy (MLD) and Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) are among the most frequent inherited leukodystrophies. X-ALD and MLD can affect both chi...
Clinicaltrials.gov 14 results
https://clinicaltrials.gov/ct2/show/NCT04687007
Mar 8th, 2023 - 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 ...
https://clinicaltrials.gov/ct2/show/NCT04675749
Mar 8th, 2023 - X-linked adrenoleukodystrophy (X-ALD) is a hereditary white matter disorder caused by mutations in the ABCD1 gene leading to disturbances in the metabolism of fatty acids. This results in an accumulation of very long chain fatty acids (VLCFA) in t...
https://clinicaltrials.gov/ct2/show/NCT05008874
Dec 13th, 2022 - 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/NCT04925349
Oct 12th, 2022 - X-linked Adrenoleukodystrophy (X-ALD), Metachromatic Leukodystrophy (MLD) and Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP) are among the most frequent inherited leukodystrophies. X-ALD and MLD can affect both chi...
https://clinicaltrials.gov/ct2/show/NCT05146284
Sep 28th, 2022 - A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL770 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN). There are 3 study periods. Screening Visit: within a maxi...
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.
