Hematopoietic Stem-Cell Transplantation in Globoid-Cell Leukodystrophy
Globoid-cell leukodystrophy is an autosomal recessive disease due to greatly diminished or absent activity of the lysosomal enzyme galactocerebrosidase. 1 The disease is characterized by the progressive loss of central and peripheral myelin and by spasticity, dementia, and peripheral neuropathy. It...
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| Veröffentlicht in: | The New England journal of medicine 1998-04, Vol.338 (16), p.1119-1127 |
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| container_title | The New England journal of medicine |
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| creator | Krivit, William Shapiro, Elsa G Peters, Charles Wagner, John E Cornu, Guy Kurtzberg, Joanne Wenger, David A Kolodny, Edwin H Vanier, Marie T Loes, Daniel J Dusenbery, Kathryn Lockman, Lawrence A |
| description | Globoid-cell leukodystrophy is an autosomal recessive disease due to greatly diminished or absent activity of the lysosomal enzyme galactocerebrosidase.
1
The disease is characterized by the progressive loss of central and peripheral myelin and by spasticity, dementia, and peripheral neuropathy. It ends in a chronic vegetative state and early death. The more common form begins in early infancy and is rapidly progressive, often leading to death within two years. The late-onset form typically begins later in childhood, has a more insidious onset, and progresses over a period of several years to death.
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Hematopoietic stem-cell transplantation has been shown to alter . . . |
| doi_str_mv | 10.1056/NEJM199804163381605 |
| format | Article |
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1
The disease is characterized by the progressive loss of central and peripheral myelin and by spasticity, dementia, and peripheral neuropathy. It ends in a chronic vegetative state and early death. The more common form begins in early infancy and is rapidly progressive, often leading to death within two years. The late-onset form typically begins later in childhood, has a more insidious onset, and progresses over a period of several years to death.
2
,
3
Hematopoietic stem-cell transplantation has been shown to alter . . .</description><identifier>ISSN: 0028-4793</identifier><identifier>EISSN: 1533-4406</identifier><identifier>DOI: 10.1056/NEJM199804163381605</identifier><identifier>PMID: 9545360</identifier><identifier>CODEN: NEJMAG</identifier><language>eng</language><publisher>Boston, MA: Massachusetts Medical Society</publisher><subject>Biological and medical sciences ; Central Nervous System Diseases - etiology ; Central Nervous System Diseases - prevention & control ; Central Nervous System Diseases - therapy ; Cerebrospinal Fluid Proteins - analysis ; Child ; Child, Preschool ; Enzymes ; Female ; Galactosylceramidase - metabolism ; Hematopoietic Stem Cell Transplantation ; Humans ; Infant ; Leukocytes ; Leukocytes - enzymology ; Leukodystrophy, Globoid Cell - complications ; Leukodystrophy, Globoid Cell - therapy ; Male ; Medical sciences ; Miscellaneous ; Neurological disorders ; Surgery (general aspects). Transplantations, organ and tissue grafts. Graft diseases ; Transplantation, Homologous ; Transplants & implants</subject><ispartof>The New England journal of medicine, 1998-04, Vol.338 (16), p.1119-1127</ispartof><rights>Copyright © 1998 Massachusetts Medical Society. All rights reserved.</rights><rights>1998 INIST-CNRS</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c625t-c216680e765b8341aa6b35057abba9a17ec61f88e70de95f243894bf78f3785d3</citedby><cites>FETCH-LOGICAL-c625t-c216680e765b8341aa6b35057abba9a17ec61f88e70de95f243894bf78f3785d3</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://www.nejm.org/doi/pdf/10.1056/NEJM199804163381605$$EPDF$$P50$$Gmms$$H</linktopdf><linktohtml>$$Uhttps://www.nejm.org/doi/full/10.1056/NEJM199804163381605$$EHTML$$P50$$Gmms$$H</linktohtml><link.rule.ids>314,776,780,2746,2747,26080,27901,27902,52357,54039</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=2206756$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/9545360$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Krivit, William</creatorcontrib><creatorcontrib>Shapiro, Elsa G</creatorcontrib><creatorcontrib>Peters, Charles</creatorcontrib><creatorcontrib>Wagner, John E</creatorcontrib><creatorcontrib>Cornu, Guy</creatorcontrib><creatorcontrib>Kurtzberg, Joanne</creatorcontrib><creatorcontrib>Wenger, David A</creatorcontrib><creatorcontrib>Kolodny, Edwin H</creatorcontrib><creatorcontrib>Vanier, Marie T</creatorcontrib><creatorcontrib>Loes, Daniel J</creatorcontrib><creatorcontrib>Dusenbery, Kathryn</creatorcontrib><creatorcontrib>Lockman, Lawrence A</creatorcontrib><title>Hematopoietic Stem-Cell Transplantation in Globoid-Cell Leukodystrophy</title><title>The New England journal of medicine</title><addtitle>N Engl J Med</addtitle><description>Globoid-cell leukodystrophy is an autosomal recessive disease due to greatly diminished or absent activity of the lysosomal enzyme galactocerebrosidase.
1
The disease is characterized by the progressive loss of central and peripheral myelin and by spasticity, dementia, and peripheral neuropathy. It ends in a chronic vegetative state and early death. The more common form begins in early infancy and is rapidly progressive, often leading to death within two years. The late-onset form typically begins later in childhood, has a more insidious onset, and progresses over a period of several years to death.
2
,
3
Hematopoietic stem-cell transplantation has been shown to alter . . .</description><subject>Biological and medical sciences</subject><subject>Central Nervous System Diseases - etiology</subject><subject>Central Nervous System Diseases - prevention & control</subject><subject>Central Nervous System Diseases - therapy</subject><subject>Cerebrospinal Fluid Proteins - analysis</subject><subject>Child</subject><subject>Child, Preschool</subject><subject>Enzymes</subject><subject>Female</subject><subject>Galactosylceramidase - metabolism</subject><subject>Hematopoietic Stem Cell Transplantation</subject><subject>Humans</subject><subject>Infant</subject><subject>Leukocytes</subject><subject>Leukocytes - enzymology</subject><subject>Leukodystrophy, Globoid Cell - complications</subject><subject>Leukodystrophy, Globoid Cell - therapy</subject><subject>Male</subject><subject>Medical sciences</subject><subject>Miscellaneous</subject><subject>Neurological disorders</subject><subject>Surgery (general aspects). Transplantations, organ and tissue grafts. Graft diseases</subject><subject>Transplantation, Homologous</subject><subject>Transplants & implants</subject><issn>0028-4793</issn><issn>1533-4406</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>1998</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><sourceid>BEC</sourceid><sourceid>BENPR</sourceid><sourceid>GUQSH</sourceid><sourceid>M2O</sourceid><recordid>eNqFkctKw0AUhgdRaq0-gQhFxI1E535ZSulFqbqwrsMkmWBqkokzyaJv75QEFyJ6Nmfxf-f2HwDOEbxFkPG75_njE1JKQoo4IRJxyA7AGDFCIkohPwRjCLGMqFDkGJx4v4UhEFUjMFKMMsLhGCxWptKtbWxh2iKdvramimamLKcbp2vflLpudVvYelrU02VpE1tkvb423YfNdr51tnnfnYKjXJfenA15At4W881sFa1flg-z-3WUcszaKMWIcwmN4CyRhCKteUIYZEIniVYaCZNylEtpBMyMYjmmRCqa5ELmREiWkQm47vs2zn52xrdxVfg07KNrYzsfCyUEYxj-CyJOEVSCB_DyB7i1navDETHGRHEa7A0Q6aHUWe-dyePGFZV2uxjBeP-L-JdfhKqLoXWXVCb7rhnMD_rVoGuf6jIPjqeF_8Ywhlyw_YY3PVZVPq7Ntvpz6BeQf5uq</recordid><startdate>19980416</startdate><enddate>19980416</enddate><creator>Krivit, William</creator><creator>Shapiro, Elsa G</creator><creator>Peters, Charles</creator><creator>Wagner, John E</creator><creator>Cornu, Guy</creator><creator>Kurtzberg, Joanne</creator><creator>Wenger, David A</creator><creator>Kolodny, Edwin H</creator><creator>Vanier, Marie T</creator><creator>Loes, Daniel J</creator><creator>Dusenbery, Kathryn</creator><creator>Lockman, Lawrence A</creator><general>Massachusetts Medical Society</general><scope>IQODW</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>0TZ</scope><scope>7RV</scope><scope>7X7</scope><scope>7XB</scope><scope>8AO</scope><scope>8C1</scope><scope>8FE</scope><scope>8FH</scope><scope>8FI</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>AN0</scope><scope>AZQEC</scope><scope>BBNVY</scope><scope>BEC</scope><scope>BENPR</scope><scope>BHPHI</scope><scope>CCPQU</scope><scope>DWQXO</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>GNUQQ</scope><scope>GUQSH</scope><scope>HCIFZ</scope><scope>K0Y</scope><scope>LK8</scope><scope>M0R</scope><scope>M0T</scope><scope>M1P</scope><scope>M2M</scope><scope>M2O</scope><scope>M2P</scope><scope>M7P</scope><scope>MBDVC</scope><scope>NAPCQ</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>PRINS</scope><scope>PSYQQ</scope><scope>Q9U</scope><scope>7T5</scope><scope>H94</scope><scope>7X8</scope></search><sort><creationdate>19980416</creationdate><title>Hematopoietic Stem-Cell Transplantation in Globoid-Cell Leukodystrophy</title><author>Krivit, William ; Shapiro, Elsa G ; Peters, Charles ; Wagner, John E ; Cornu, Guy ; Kurtzberg, Joanne ; Wenger, David A ; Kolodny, Edwin H ; Vanier, Marie T ; Loes, Daniel J ; Dusenbery, Kathryn ; Lockman, Lawrence A</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c625t-c216680e765b8341aa6b35057abba9a17ec61f88e70de95f243894bf78f3785d3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>1998</creationdate><topic>Biological and medical sciences</topic><topic>Central Nervous System Diseases - etiology</topic><topic>Central Nervous System Diseases - prevention & control</topic><topic>Central Nervous System Diseases - therapy</topic><topic>Cerebrospinal Fluid Proteins - analysis</topic><topic>Child</topic><topic>Child, Preschool</topic><topic>Enzymes</topic><topic>Female</topic><topic>Galactosylceramidase - metabolism</topic><topic>Hematopoietic Stem Cell Transplantation</topic><topic>Humans</topic><topic>Infant</topic><topic>Leukocytes</topic><topic>Leukocytes - enzymology</topic><topic>Leukodystrophy, Globoid Cell - complications</topic><topic>Leukodystrophy, Globoid Cell - therapy</topic><topic>Male</topic><topic>Medical sciences</topic><topic>Miscellaneous</topic><topic>Neurological disorders</topic><topic>Surgery (general aspects). 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1
The disease is characterized by the progressive loss of central and peripheral myelin and by spasticity, dementia, and peripheral neuropathy. It ends in a chronic vegetative state and early death. The more common form begins in early infancy and is rapidly progressive, often leading to death within two years. The late-onset form typically begins later in childhood, has a more insidious onset, and progresses over a period of several years to death.
2
,
3
Hematopoietic stem-cell transplantation has been shown to alter . . .</abstract><cop>Boston, MA</cop><pub>Massachusetts Medical Society</pub><pmid>9545360</pmid><doi>10.1056/NEJM199804163381605</doi><tpages>9</tpages><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0028-4793 |
| ispartof | The New England journal of medicine, 1998-04, Vol.338 (16), p.1119-1127 |
| issn | 0028-4793 1533-4406 |
| language | eng |
| recordid | cdi_proquest_miscellaneous_79775520 |
| source | MEDLINE; Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals; New England Journal of Medicine |
| subjects | Biological and medical sciences Central Nervous System Diseases - etiology Central Nervous System Diseases - prevention & control Central Nervous System Diseases - therapy Cerebrospinal Fluid Proteins - analysis Child Child, Preschool Enzymes Female Galactosylceramidase - metabolism Hematopoietic Stem Cell Transplantation Humans Infant Leukocytes Leukocytes - enzymology Leukodystrophy, Globoid Cell - complications Leukodystrophy, Globoid Cell - therapy Male Medical sciences Miscellaneous Neurological disorders Surgery (general aspects). Transplantations, organ and tissue grafts. Graft diseases Transplantation, Homologous Transplants & implants |
| title | Hematopoietic Stem-Cell Transplantation in Globoid-Cell Leukodystrophy |
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