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About 1,015 results

ALLMedicine™ Adrenoleukodystrophy Center

Research & Reviews  335 results

Leriglitazone: frustration and hope in adrenoleukodystrophy.
https://doi.org/10.1016/S1474-4422(22)00518-X
The Lancet. Neurology; Schöls L

Jan 22nd, 2023 - Leriglitazone: frustration and hope in adrenoleukodystrophy.|2023|Schöls L,|

Safety and efficacy of leriglitazone for preventing disease progression in men with adr...
https://doi.org/10.1016/S1474-4422(22)00495-1
The Lancet. Neurology; Köhler W, Engelen M et. al.

Jan 22nd, 2023 - Adult patients with adrenoleukodystrophy have a poor prognosis owing to development of adrenomyeloneuropathy. Additionally, a large proportion of patients with adrenomyeloneuropathy develop life-threatening progressive cerebral adrenoleukodystroph...

A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant ...
https://clinicaltrials.gov/ct2/show/NCT04528706

Jan 10th, 2023 - An Open-Label, multicenter study in male pediatric patients with cerebral x-linked adrenoleukodystrophy (cald) to assess the effects of MIN-102 treatment on disease progression prior to human stem cell transplant (HSCT)

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

Dec 21st, 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 ...

Saturated very long-chain fatty acids regulate macrophage plasticity and invasiveness.
https://doi.org/10.1186/s12974-022-02664-y
Journal of Neuroinflammation; Zierfuss B, Buda A et. al.

Dec 18th, 2022 - Saturated very long-chain fatty acids (VLCFA, ≥ C22), enriched in brain myelin and innate immune cells, accumulate in X-linked adrenoleukodystrophy (X-ALD) due to inherited dysfunction of the peroxisomal VLCFA transporter ABCD1. In its severest fo...

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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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Drugs  1 results see all →

Clinicaltrials.gov  36 results

A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant ...
https://clinicaltrials.gov/ct2/show/NCT04528706

Jan 10th, 2023 - An Open-Label, multicenter study in male pediatric patients with cerebral x-linked adrenoleukodystrophy (cald) to assess the effects of MIN-102 treatment on disease progression prior to human stem cell transplant (HSCT)

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

Dec 21st, 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

Dec 13th, 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...

Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
https://clinicaltrials.gov/ct2/show/NCT02698579

Nov 8th, 2022 - This is a multi-center, long-term safety and efficacy follow-up study for participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product in a parent clinical study. After completing a parent clinical study (approxima...

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

Oct 12th, 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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News  23 results

FDA Advisors Cleared a Path for New Gene Therapies in 2022
https://www.medpagetoday.com/hematologyoncology/othercancers/102452

Dec 30th, 2022 - Earlier this year, MedPage Today reported on an FDA advisory committee meeting in which it was unanimously recommended that two gene therapies be approved -- betibeglogene autotemcel (beti-cel, Zynteglo) for patients with transfusion-dependent bet...

Early Win for Gene Therapy in Rare Form of SCID
https://www.medpagetoday.com/genetics/generalgenetics/102358

Dec 21st, 2022 - A gene therapy for a rare type of severe combined immunodeficiency (SCID) was safe and sufficiently restored T-cell immunity in an early trial, results from a phase I/II study showed. At a single center, all 10 infants with Artemis-deficient SCID ...

Gene Therapy for Degenerative Brain Disorder Wins FDA Nod
https://www.medpagetoday.com/neurology/generalneurology/100808

Sep 19th, 2022 - The FDA granted accelerated approval to elivaldogene autotemcel (eli-cel; Skysona), a gene therapy to treat early, active cerebral adrenoleukodystrophy (CALD) in boys ages 4 to 17 years, drugmaker bluebird bio announced Friday. Childhood CALD is a...

FDA Fast Tracks Approval of Novel Therapy for Rare Neurologic Disorder
https://www.medscape.com/viewarticle/981025

Sep 19th, 2022 - The US Food and Drug Administration (FDA) has granted accelerated approval to the novel gene therapy elivaldogene autotemcel (Skysona, bluebird bio) for the treatment of cerebral adrenoleukodystrophy (CALD). The therapy "is indicated to slow the p...

FDA Committee Unanimous on Two Gene Therapies
https://www.medscape.com/viewarticle/975467

Jun 13th, 2022 - A US Food and Drug Administration advisory committee gave a unanimous thumbs up to two new gene therapies: betibeglogene autotemcel (beti-cel) for the treatment of a blood disorder, transfusion-dependent beta-thalassemia; and elivaldogene autotemc...

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Patient Education  9 results see all →