ALLMedicine™ Adrenoleukodystrophy Center

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...

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 ...

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...

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...

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...

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...

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...

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...

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 ...

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...

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...

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...

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...

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 ...

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 ...

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.

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.