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About 798 results

ALLMedicine™ Adrenoleukodystrophy Center

Research & Reviews  284 results

Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)
https://clinicaltrials.gov/ct2/show/NCT05200104

Jan 20th, 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...

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

Jan 19th, 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 ...

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

Jan 19th, 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...

Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)
https://clinicaltrials.gov/ct2/show/NCT05146284

Dec 6th, 2021 - 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...

Adult-onset adrenoleukodystrophy presenting with status epilepticus and psychosis.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8634231
BMJ Case Reports; Mehta AM, Prabhu M et. al.

Dec 2nd, 2021 - Adrenoleukodystrophy (ALD) is an X linked recessive genetic disorder caused by an abnormality in the ABCD1 gene on the X chromosome, that affects 1 in 20 000 people. In X linked adrenoleukodystrophy (X-ALD), a defect in lignoceroyl-coenzyme A liga...

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Guidelines  2 results

MRI surveillance of boys with X-linked adrenoleukodystrophy identified by newborn scree...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8113077
Journal of Inherited Metabolic Disease; Mallack EJ, Turk BR et. al.

Dec 30th, 2020 - Among boys with X-Linked adrenoleukodystrophy, a subset will develop childhood cerebral adrenoleukodystrophy (CCALD). CCALD is typically lethal without hematopoietic stem cell transplant before or soon after symptom onset. We sought to establish e...

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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Clinicaltrials.gov  19 results

Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)
https://clinicaltrials.gov/ct2/show/NCT05200104

Jan 20th, 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...

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

Jan 19th, 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 ...

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

Jan 19th, 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...

Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)
https://clinicaltrials.gov/ct2/show/NCT05146284

Dec 6th, 2021 - 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...

A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
https://clinicaltrials.gov/ct2/show/NCT01896102

Sep 21st, 2021 - This trial will assess the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector, for the treatment of cerebral adrenoleukodystrophy (CALD). A participan...

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News  9 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...

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 ...

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 ...

Gene therapy for cerebral adrenoleukodystrophy shows promise
https://www.mdedge.com/neurology/article/149021/pediatrics/gene-therapy-cerebral-adrenoleukodystrophy-shows-promise
Bianca Nogrady

Oct 9th, 2017 - Gene therapy using autologous hematopoietic stem cells appears safe and effective as a treatment for early-stage cerebral adrenoleukodystrophy, according to an interim analysis of results from the STARBEAM study. X-linked adrenoleukodystrophy is c.

Gene therapy for cerebral adrenoleukodystrophy shows promise
https://www.mdedge.com/pediatrics/article/149021/pediatrics/gene-therapy-cerebral-adrenoleukodystrophy-shows-promise
Bianca Nogrady

Oct 9th, 2017 - Gene therapy using autologous hematopoietic stem cells appears safe and effective as a treatment for early-stage cerebral adrenoleukodystrophy, according to an interim analysis of results from the STARBEAM study. X-linked adrenoleukodystrophy is c.

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Patient Education  5 results see all →