ALLMedicine™ Adrenoleukodystrophy Center
Research & Reviews 713 results
https://doi.org/10.1515/jpem-2020-0652
Journal of Pediatric Endocrinology & Metabolism : JPEM; Zhu J, Breault DT
Apr 5th, 2021 - X-linked adrenoleukodystrophy (ALD) affects up to 25% of boys diagnosed with adrenal insufficiency in childhood. Because early identification of these individuals can be lifesaving, all boys with new-onset primary adrenal insufficiency should be t...
https://doi.org/10.1089/hum.2021.053
Human Gene Therapy; Bougneres P, Hacein-Bey-Abina S et. al.
Apr 1st, 2021 - BACKGROUND In 2009, cerebral adrenoleukodystrophy (c-ALD) became the first brain disease to be treated with lentiviral-based hematopoietic stem cell gene therapy with the ABCD1 gene in four boys (P1-P4) who had demyelinating lesions expected to be...
https://doi.org/10.1111/jon.12844
Journal of Neuroimaging : Official Journal of the America... Ozturk K, Nascene D
Mar 30th, 2021 - To determine whether cerebral adrenoleukodystrophy (cALD) or brain irradiation in patients with primary brain tumor affects T1-weighted imaging (T1WI) signal intensity (SI) of the dentate nucleus (DN) in a pediatric cohort who had received consecu...
https://doi.org/10.1111/petr.14015
Pediatric Transplantation; Yalcin K, Çelen SS et. al.
Mar 29th, 2021 - ALD is a rare X-linked peroxisomal metabolic disorder with many distinct phenotypes of disease that emerge on a wide scale from adrenal insufficiency to fatal cALD which progresses to a vegetative state within a few years. Currently, HSCT is the o...
https://doi.org/10.1038/s41467-021-22114-2 10.3390/ijns2040015 10.1038/361726a0 10.1074/jbc.M112.445445 10.1016/j.bbamcr.2006.07.010 10.1093/jnen/59.2.89 10.1093/brain/awy299 10.1016/j.jpeds.2004.10.067 10.1097/00005072-199211000-00007 10.1093/brain/awy127 10.1093/brain/awv250 10.1016/j.bbmt.2018.09.036 10.1126/science.1171242 10.1056/NEJMoa1700554 10.1002/jdn.10003 10.3174/ajnr.A4500 10.1038/s41582-018-0058-z 10.3389/fneur.2019.00338 10.1001/jamaneurol.2019.2137 10.3389/fneur.2018.01167 10.1038/s41591-018-0304-3 10.1016/j.ebiom.2015.11.036 10.1016/j.brainres.2017.05.011 10.1177/1352458517715132 10.1371/journal.pone.0032218 10.1007/s12031-011-9672-6 10.1016/j.jneuroim.2015.11.021 10.1385/JMN:13:3:247 10.1136/jnnp-2018-318704 10.3348/kjr.2018.0356 10.1177/1352458518814117 10.1001/jamaneurol.2019.2399 10.1212/NXI.0000000000000598 10.1182/blood-2018-11-887240 10.1186/1471-2377-14-58 10.1212/WNL.0000000000008572 10.1002/acn3.51188
Nature Communications; Weinhofer I, Rommer P et. al.
Mar 23rd, 2021 - X-linked adrenoleukodystrophy (X-ALD), the most frequent monogenetic disorder of brain white matter, is highly variable, ranging from slowly progressive adrenomyeloneuropathy (AMN) to life-threatening inflammatory brain demyelination (CALD). In th...
Guidelines 1 results
https://doi.org/10.1210/jc.2018-00920
The Journal of Clinical Endocrinology and Metabolism; Regelmann MO, Kamboj MK et. al.
Oct 5th, 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...
Clinicaltrials.gov 739 results
https://doi.org/10.1515/jpem-2020-0652
Journal of Pediatric Endocrinology & Metabolism : JPEM; Zhu J, Breault DT
Apr 5th, 2021 - X-linked adrenoleukodystrophy (ALD) affects up to 25% of boys diagnosed with adrenal insufficiency in childhood. Because early identification of these individuals can be lifesaving, all boys with new-onset primary adrenal insufficiency should be t...
https://doi.org/10.1089/hum.2021.053
Human Gene Therapy; Bougneres P, Hacein-Bey-Abina S et. al.
Apr 1st, 2021 - BACKGROUND In 2009, cerebral adrenoleukodystrophy (c-ALD) became the first brain disease to be treated with lentiviral-based hematopoietic stem cell gene therapy with the ABCD1 gene in four boys (P1-P4) who had demyelinating lesions expected to be...
https://doi.org/10.1111/jon.12844
Journal of Neuroimaging : Official Journal of the America... Ozturk K, Nascene D
Mar 30th, 2021 - To determine whether cerebral adrenoleukodystrophy (cALD) or brain irradiation in patients with primary brain tumor affects T1-weighted imaging (T1WI) signal intensity (SI) of the dentate nucleus (DN) in a pediatric cohort who had received consecu...
https://doi.org/10.1111/petr.14015
Pediatric Transplantation; Yalcin K, Çelen SS et. al.
Mar 29th, 2021 - ALD is a rare X-linked peroxisomal metabolic disorder with many distinct phenotypes of disease that emerge on a wide scale from adrenal insufficiency to fatal cALD which progresses to a vegetative state within a few years. Currently, HSCT is the o...
https://doi.org/10.1038/s41467-021-22114-2 10.3390/ijns2040015 10.1038/361726a0 10.1074/jbc.M112.445445 10.1016/j.bbamcr.2006.07.010 10.1093/jnen/59.2.89 10.1093/brain/awy299 10.1016/j.jpeds.2004.10.067 10.1097/00005072-199211000-00007 10.1093/brain/awy127 10.1093/brain/awv250 10.1016/j.bbmt.2018.09.036 10.1126/science.1171242 10.1056/NEJMoa1700554 10.1002/jdn.10003 10.3174/ajnr.A4500 10.1038/s41582-018-0058-z 10.3389/fneur.2019.00338 10.1001/jamaneurol.2019.2137 10.3389/fneur.2018.01167 10.1038/s41591-018-0304-3 10.1016/j.ebiom.2015.11.036 10.1016/j.brainres.2017.05.011 10.1177/1352458517715132 10.1371/journal.pone.0032218 10.1007/s12031-011-9672-6 10.1016/j.jneuroim.2015.11.021 10.1385/JMN:13:3:247 10.1136/jnnp-2018-318704 10.3348/kjr.2018.0356 10.1177/1352458518814117 10.1001/jamaneurol.2019.2399 10.1212/NXI.0000000000000598 10.1182/blood-2018-11-887240 10.1186/1471-2377-14-58 10.1212/WNL.0000000000008572 10.1002/acn3.51188
Nature Communications; Weinhofer I, Rommer P et. al.
Mar 23rd, 2021 - X-linked adrenoleukodystrophy (X-ALD), the most frequent monogenetic disorder of brain white matter, is highly variable, ranging from slowly progressive adrenomyeloneuropathy (AMN) to life-threatening inflammatory brain demyelination (CALD). In th...
News 13 results
https://www.medscape.com/viewarticle/947868
Mar 21st, 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...
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.
https://www.medscape.com/viewarticle/886974
Oct 11th, 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 ...
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.
https://www.medpagetoday.com/neurology/generalneurology/68321
Oct 4th, 2017 - Action Points Note that this phase II/III trial of autologous stem cell transplant with gene therapy in boys with adrenoleukodystrophy showed promising efficacy results. Be aware that the new treatment was not compared directly to the standard of ...