ALLMedicine™ Adrenomyeloneuropathy Center
Research & Reviews 65 results
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://www.ncbi.nlm.nih.gov/pmc/articles/PMC9542479
Neurotherapeutics : the Journal of the American Society F... Nemeth CL, Gӧk Ö et. al.
Oct 8th, 2022 - X-linked adrenoleukodystrophy (ALD) is a genetic disorder that presents neurologically as either a rapid and fatal cerebral demyelinating disease in childhood (childhood cerebral adrenoleukodystrophy; ccALD) or slow degeneration of the spinal cord...
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...
https://clinicaltrials.gov/ct2/show/NCT05200104
Sep 28th, 2022 - A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL065 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN). There will be a total of 3 study periods. Screening perio...
https://clinicaltrials.gov/ct2/show/NCT04687007
Aug 17th, 2022 - 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 ...
Clinicaltrials.gov 14 results
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...
https://clinicaltrials.gov/ct2/show/NCT05200104
Sep 28th, 2022 - A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL065 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN). There will be a total of 3 study periods. Screening perio...
https://clinicaltrials.gov/ct2/show/NCT04687007
Aug 17th, 2022 - 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/NCT05394064
Jun 2nd, 2022 - The study consists of two parts after infusion of SBT101: Part 1: A blinded 24-month core study period to evaluate the safety and potential impact of SBT101 on disease progression. Part 1 will consist of 2 phases: Phase 1: Dose-Escalation Phase: T...
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.
