ArticleMolecular genetics and metabolism2015
Disease specific therapies in leukodystrophies and leukoencephalopathies.
Article in Molecular genetics and metabolism, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
25 citing papers in PubMed, 1 synthesis or guideline pooled it, 54 citations in OpenAlex.
- Revised consensus statement on the preventive and symptomatic care of patients with leukodystrophies.Molecular genetics and metabolism · 2017Guideline
- Randomized Clinical Trial of First-Line Genome Sequencing in Pediatric White Matter Disorders.Annals of neurology · 2020Trial
- Preventing Parkinson's disease in the context of movement disorders: a narrative review of current evidence and future directions.Neurological research and practice · 2026Review
- Retinal Vasculopathy With Cerebral Leukoencephalopathy Mimicking a Brain Tumor: A Case Report.Cureus · 2026Article
- Death Causes Among Iranian Children With Leukodystrophies.Journal of child neurology · 2025Article
- Remyelination in the Central Nervous System.Cold Spring Harbor perspectives in biology · 2024Review
- Approaches to diagnosis for individuals with a suspected inherited white matter disorder.Handbook of clinical neurology · 2024Review
- Neural Cells for Neurodegenerative Diseases in Clinical Trials.Stem cells translational medicine · 2023Review
- Cortical interneuron development is affected in 4H leukodystrophy.Brain : a journal of neurology · 2023Article
- Applications of Induced Pluripotent Stem Cell-Derived Glia in Brain Disease Research and Treatment.Handbook of experimental pharmacology · 2023Review
- Gene supplementation ofMolecular therapy. Methods & clinical development · 2021Article
- The LORIS MyeliNeuroGene rare disease database for natural history studies and clinical trial readiness.Orphanet journal of rare diseases · 2021Article
- Genetic disorders with central nervous system white matter abnormalities: An update.Clinical genetics · 2021Review
- Practical Approaches and Knowledge Gaps in the Care for Children With Leukodystrophies.Journal of child neurology · 2021Review
- Natural history of multiple sulfatase deficiency: Retrospective phenotyping and functional variant analysis to characterize an ultra-rare disease.Journal of inherited metabolic disease · 2020Article
- Novel and known morbidities of leukodystrophies identified using a phenome-wide association study.Neurology. Clinical practice · 2020Article
- Failure of intrathecal allogeneic mesenchymal stem cells to halt progressive demyelination in two boys with cerebral adrenoleukodystrophy.Stem cells translational medicine · 2020Article
- POLR3-Related Leukodystrophy: Exploring Potential Therapeutic Approaches.Frontiers in cellular neuroscience · 2020Article
- Long-Term Safety, Immunologic Response, and Imaging Outcomes following Neural Stem Cell Transplantation for Pelizaeus-Merzbacher Disease.Stem cell reports · 2019Article
- Adulthood leukodystrophies.Nature reviews. Neurology · 2018Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
24 authors at 18 institutions in 4 countries.
Funding
Abstract
Leukodystrophies are a heterogeneous, often progressive group of disorders manifesting a wide range of symptoms and complications. Most of these disorders have historically had no etiologic or disease specific therapeutic approaches. Recently, a greater understanding of the pathologic mechanisms associated with leukodystrophies has allowed clinicians and researchers to prioritize treatment strategies and advance research in therapies for specific disorders, some of which are on the verge of pilot or Phase I/II clinical trials. This shifts the care of leukodystrophy patients from the management of the complex array of symptoms and sequelae alone to targeted therapeutics. The unmet needs of leukodystrophy patients still remain an overwhelming burden. While the overwhelming consensus is that these disorders collectively are symptomatically treatable, leukodystrophy patients are in need of advanced therapies and if possible, a cure.
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Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.