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About 755 results

ALLMedicine™ Adrenoleukodystrophy Center

Research & Reviews  269 results

Population Pharmacokinetic Analysis of N-acetylcysteine in Pediatric Patients with Inhe...
https://doi.org/10.1002/jcph.1943
Journal of Clinical Pharmacology; Sahasrabudhe SA, Kartha RV et. al.

Jul 19th, 2021 - N-acetylcysteine (NAC) has been utilized in patients with cerebral adrenoleukodystrophy(cALD) as an antioxidant agent in association with hematopoietic stem cell transplant (HSCT). However, an understanding of the pharmacokinetic characteristics o...

Endocrine dysfunction in adrenoleukodystrophy.
https://doi.org/10.1016/B978-0-12-819973-2.00018-6
Handbook of Clinical Neurology; Engelen M, Kemp S et. al.

Jul 17th, 2021 - X-linked adrenoleukodystrophy (ALD) is a peroxisomal disorder caused by mutations in the ABCD1 gene and characterized by impaired very long-chain fatty acid beta-oxidation. Clinically, male patients develop adrenal failure and a progressive myelop...

Modulation of mitochondrial and inflammatory homeostasis through RIP140 is neuroprotect...
https://doi.org/10.1111/nan.12747
Neuropathology and Applied Neurobiology; Ranea-Robles P, Galino J et. al.

Jul 9th, 2021 - Mitochondrial dysfunction and inflammation are at the core of axonal degeneration in several multifactorial neurodegenerative diseases, including multiple sclerosis, Alzheimer's and Parkinson's disease. The transcriptional coregulator RIP140/NRIP1...

Imaging in X-Linked Adrenoleukodystrophy.
https://doi.org/10.1055/s-0041-1730937
Neuropediatrics van de Stadt SIW, Huffnagel IC et. al.

Jul 1st, 2021 - Magnetic resonance imaging (MRI) is the gold standard for the detection of cerebral lesions in X-linked adrenoleukodystrophy (ALD). ALD is one of the most common peroxisomal disorders and is characterized by a defect in degradation of very long ch...

Retrospective evaluation of patients with X-linked adrenoleukodystrophy with a wide ran...
https://doi.org/10.1515/jpem-2021-0032
Journal of Pediatric Endocrinology & Metabolism : JPEM; Olgac A, Kasapkara ÇS et. al.

Jun 24th, 2021 - X-linked adrenoleukodystrophy (X-ALD), is a peroxisomal inborn error of metabolism caused due to the loss of function variants of ABCD1 gene that leads to accumulation of very long chain fatty acids (VLCFAs) in several tissues including the neurol...

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Guidelines  1 results

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  16 results

A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy
https://clinicaltrials.gov/ct2/show/NCT02595489

Nov 14th, 2019 - Prior research suggests that higher vitamin D levels in the blood are associated with reduced brain inflammation among individuals with multiple sclerosis, a disease that is similar to the cerebral demyelinating form of ALD. However, serious side ...

Lentiviral Gene Therapy for X-ALD
https://clinicaltrials.gov/ct2/show/NCT03727555

Sep 19th, 2019 - X-linked adrenoleukodystrophy (X-ALD) is a devastating neurological disorder caused by mutations in the ABCD1 gene that encodes a peroxisomal ATP-binding cassette transporter (ABCD1). ABCD1 is responsible for transport of CoA-activated very long-c...

HSCT for High Risk Inherited Inborn Errors
https://clinicaltrials.gov/ct2/show/NCT00383448

Jul 11th, 2019 - Hematopoietic stem cell transplantation has proven effective therapy for individuals with adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD, or Krabbe disease). However, for patients with advanced o...

A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.
https://clinicaltrials.gov/ct2/show/NCT03231878

Mar 25th, 2019 - This is a Phase II/III, randomized, double-blind, placebo-controlled, multicenter, two parallel-group study in male patients with the AMN phenotype of X-linked adrenoleukodystrophy (X-ALD) to assess the efficacy and safety of MIN-102 treatment. St...

Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy
https://clinicaltrials.gov/ct2/show/NCT01594853

Aug 29th, 2018 - X-linked adrenoleukodystrophy (X-ALD), a [sex-linked] progressive neurodegenerative disease, is caused by a defect in the ABCD1 gene. The disease is expressed in multiple ways, but the most common adult form is adrenomyeloneuropathy (AMN), which r...

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News  11 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...

Neurocognitive Decline and Motor Regression in a 34-Year-Old Woman
https://reference.medscape.com/viewarticle/845434_3

Apr 23rd, 2018 - Discussion This patient's radiologic data and clinical picture were consistent with metachromatic leukodystrophy (MLD). Because the patient had already progressed to a bedridden, quadriparetic state, she was referred for rehabilitation, and the fa...

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 ...

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.

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 ...

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Patient Education  5 results see all →