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About 198 results

ALLMedicine™ Adrenomyeloneuropathy Center

Research & Reviews  64 results

A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)
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...

SMART-ALD - A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD)
https://clinicaltrials.gov/ct2/show/NCT04687007

May 25th, 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 ...

Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia
https://clinicaltrials.gov/ct2/show/NCT05008874

May 17th, 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...

Therapeutic potential of deuterium-stabilized (R)-pioglitazone-PXL065-for X-linked adre...
https://doi.org/10.1002/jimd.12510
Journal of Inherited Metabolic Disease; Monternier PA, Singh J et. al.

May 6th, 2022 - X-linked adrenoleukodystrophy (ALD) results from ABCD1 gene mutations which impair Very Long Chain Fatty Acids (VLCFA; C26:0 and C24:0) peroxisomal import and β-oxidation, leading to accumulation in plasma and tissues. Excess VLCFA drives impaired...

Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
https://clinicaltrials.gov/ct2/show/NCT04925349

May 6th, 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...

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Clinicaltrials.gov  14 results

A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)
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...

SMART-ALD - A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD)
https://clinicaltrials.gov/ct2/show/NCT04687007

May 25th, 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 ...

Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia
https://clinicaltrials.gov/ct2/show/NCT05008874

May 17th, 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...

Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
https://clinicaltrials.gov/ct2/show/NCT04925349

May 6th, 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...

Quality of Life in Women With X-linked Adrenoleukodystrophy
https://clinicaltrials.gov/ct2/show/NCT04675749

Apr 12th, 2022 - 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...

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News  1 results

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.

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