The DOI matches its Crossref record; 1 of the 1 references it cites were found in a registry or an index.
Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation
Abstract
Summary: Neuropsychological assessment is essential in providing documentation of the untreated natural history of storage diseases associated with dementia and quantifying the effectiveness of treatment on central nervous system function. Baseline characterization and outcome of bone marrow transplantation (BMT) for three leukodystrophies and three mucopolysaccharidoses are presented. Results suggests that BMT for Hurler syndrome, adrenoleukodystrophy, and globoid cell leukodystrophy can be effective in preventing dementia if done early enough in the disease. Sanfilippo and Hunter syndromes do not benefit and BMT is not recommended. For metachromatic leukodystrophy, BMT is not recommended for symptomatic early‐onset forms of the disease. Further longitudinal follow‐up is needed to determine whether the benefits outweigh the risks of BMT for late‐onset and preclinical metachromatic leukodystrophy.
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Citations
Cites
Adrenoleukodystrophy: Phenotypic variability and implications for therapy
Moser, H. W., Moser, A. B., Smith, K. D., Bergin, A., Borel, J., Shankroff, J., Stine, O. C., Merette, C., Ott, J., Krivit, W., et al. (1992) · Journal of Inherited Metabolic Disease · doi:10.1007/bf01799621 · Cited by 239
Self-citation
Cited by
Cord-Blood Transplants from Unrelated Donors in Patients with Hurler's Syndrome
Staba, S. L., Escolar, M. L., Poe, M., Kim, Y., Martin, P. L., Szabolcs, P., Allison-Thacker, J., Wood, S., Wenger, D. A., Rubinstein, P., et al. (2004) · New England Journal of Medicine · doi:10.1056/nejmoa032613 · Cited by 478
Self-citationShapiro, E., Krivit, W., Lockman, L., Jambaqué, I., Peters, C., Cowan, M., Harris, R., Blanche, S., Bordigoni, P., Loes, D., et al. (2000) · The Lancet · doi:10.1016/s0140-6736(00)02629-5 · Cited by 329
Self-citationKrivit, W., Peters, C., & Shapiro, E. G. (1999) · Current Opinion in Neurology · doi:10.1097/00019052-199904000-00007 · Cited by 273
Self-citationHeilbronner, R. L., Sweet, J. J., Attix, D. K., Krull, K. R., Henry, G. K., & Hart, R. P. (2010) · The Clinical Neuropsychologist · doi:10.1080/13854046.2010.526785 · Cited by 199
X linked adrenoleukodystrophy: clinical presentation, diagnosis, and therapy
van Geel, B. M., Assies, J., Wanders, R. J. A., & Barth, P. G. (1997) · Journal of Neurology, Neurosurgery & Psychiatry · doi:10.1136/jnnp.63.1.4 · Cited by 162
Metadata
- Authors
- Shapiro, E. G., Lockman, L. A., Balthazor, M., & Krivit, W.
- Venue
- Journal of Inherited Metabolic Disease
- Publisher
- Wiley
- Published
- 1995-07
- Volume
- 18
- Issue
- 4
- Pages
- 413-429
- ISSN
- 0141-8955 (ISSN-L)
- 1573-2665
- OpenAlex
- W2024990739
- Cited by
- 245
- References
- 1
- FWCI
- 5.91
- Type
- journal article
- Language
- en
- Open access
- closed
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- CoChat. (2026). Citation check: Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation. https://app.cochat.ai/papers/10.1007/bf00710053
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- Shapiro, E. G., Lockman, L. A., Balthazor, M., & Krivit, W. (1995). Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation. Journal of Inherited Metabolic Disease. https://doi.org/10.1007/bf00710053
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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