Zobrazeno 1 - 10
of 39
pro vyhledávání: '"Lawrence A. Lockman"'
Publikováno v:
Cell Transplantation, Vol 4 (1995)
Treatment and potential cure of lysosomal and peroxisomal diseases, heretofore considered fatal, has become a reality during the past decade. Bone marrow transplantation, (BMT), has provided a method for replacement of the disease-causing enzyme defi
Externí odkaz:
https://doaj.org/article/5f66ae6f54704237801eecc377605b59
Autor:
Lawrence A. Lockman
Publikováno v:
Pediatric Neurology. 122:110-112
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::775a28fd6be8d4228c0e20e907ecedbf
https://doi.org/10.1016/j.pediatrneurol.2021.05.012
https://doi.org/10.1016/j.pediatrneurol.2021.05.012
Autor:
Paul J. Orchard, Elsa Shapiro, Frank L. Rimell, Satkiran S. Grewal, Charles Peters, Stella M. Davies, John E. Wagner, Kathleen A. Delaney, S Abel, Lawrence R. Charnas, William Krivit, Alfred C. Grovas, David A. Wenger, Lawrence A. Lockman
Publikováno v:
The Journal of Pediatrics. 144:569-573
Objectives To study the efficacy of hematopoietic stem cell transplantation (HCT) for ameliorating the clinical manifestations of α-mannosidosis. Study design Four patients with α-mannosidosis underwent allogeneic HCT at the University of Minnesota
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::49bc8b97f94a5db3917a4f094a6a5646
https://doi.org/10.1016/j.jpeds.2004.01.025
https://doi.org/10.1016/j.jpeds.2004.01.025
Autor:
Elsa Shapiro, S Abel, Lawrence A. Lockman, Daniel J. Loes, Patrick R. Aubourg, Lawrence R. Charnas, Kathryn E. Dusenbery, Norma K.C. Ramsay, Paul J. Orchard, William Krivit, Shunichi Kato, Ye Tan, Charles Peters, Hugo W. Moser, Anne I. Goldman, Richard S. Ziegler, Todd E. DeFor, Satkiran S. Grewal
Publikováno v:
Blood. 104(3)
Cerebral X-linked adrenoleukodystrophy (X-ALD) is a disorder of very-long-chain fatty acid metabolism, adrenal insufficiency, and cerebral demyelination. Death occurs within 2 to 5 years of clinical onset without hematopoietic cell transplantation (H
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2ecee05366e5ce60587d247ea6d8078d
https://pubmed.ncbi.nlm.nih.gov/15073029
https://pubmed.ncbi.nlm.nih.gov/15073029
Autor:
Satkiran S. Grewal, S Abel, Todd E. DeFor, Lawrence A. Lockman, Elsa Shapiro, Paul J. Orchard, Charles Peters, William Krivit, Richard S. Ziegler, Kathryn E. Dusenbery
Publikováno v:
Bone marrow transplantation. 29(6)
Hurler syndrome (HS) is an autosomal recessive, inherited metabolic storage disorder due to deficiency of lysosomal alpha-L-iduronidase (IDU) enzyme activity. Untreated patients develop progressive mental retardation and multisystem morbidity with a
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f74fcb3e64fae9cb1bd583d1cc0c5bae
https://pubmed.ncbi.nlm.nih.gov/11960268
https://pubmed.ncbi.nlm.nih.gov/11960268
Autor:
Daniel J. Loes, Lawrence A. Lockman, Stéphane Blanche, Isabelle Jambaqué, M. Crittenden, Patrick Aubourg, Richard E. Harris, Elsa Shapiro, Morton J. Cowan, D. Ris, Richard S. Ziegler, Charles Peters, Pierre Bordigoni, Hugo W. Moser, William Krivit, B. Berg, Alain Fischer, Christopher Cox
Publikováno v:
Lancet (London, England). 356(9231)
Summary Background The childhood-onset cerebral form of X-linked adrenoleukodystrophy, a demyelinating disorder of the central nervous system, leads to a vegetative state and death within 3–5 years once clinical symptoms are detectable. The hypothe
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::54c325a1c65a2b89b5cb7732f8603392
https://pubmed.ncbi.nlm.nih.gov/11075798
https://pubmed.ncbi.nlm.nih.gov/11075798
Autor:
Elsa Shapiro, Guy Cornu, John E. Wagner, Edwin H. Kolodny, Joanne Kurtzberg, Kathryn E. Dusenbery, Lawrence A. Lockman, Daniel J. Loes, William Krivit, Charles Peters, David A. Wenger, Marie T. Vanier
Publikováno v:
The New England journal of medicine. 338(16)
BACKGROUND: Globoid-cell leukodystrophy is caused by a deficiency of galactocerebrosidase, which results in progressive central nervous system deterioration. We investigated whether allogeneic hematopoietic stem-cell transplantation can provide a sou
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::45a0047937a3db49c69e8632f9311e5c
https://pubmed.ncbi.nlm.nih.gov/9545360
https://pubmed.ncbi.nlm.nih.gov/9545360
Autor:
Nancy Bunin, Charles Peters, Craig Kollman, William Krivit, Howard J. Weinstein, Janet D. Hegland, Roberta King, Elsa Shapiro, Carl Lenarsky, Morton J. Cowan, James Anderson, John Wagner, Lawrence A. Lockman, Thomas E. Williams, Phyllis Warkentin, Jean E. Sanders, Richard R. Harris, Tom Bowen, Valerie A. Cool, Jean Henslee-Downey, Paige Kaplan, Kathryn Dusenbery, Michael E. Trigg, Leonard Sender, Peter Falk, Seymour Packman, Mary R. Crittenden, Michael Balthazor, Guy H. Grayson
Publikováno v:
Blood. 87(11)
Long-term survival and improved neuropsychological function have occurred in selected children with Hurler syndrome (MPS I H) after successful engraftment with genotypically matched sibling bons marrow transplantation (BMT). However, because few chil
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4dc353a6d9a40ea81e29c7a2919ac5ae
https://pubmed.ncbi.nlm.nih.gov/8639864
https://pubmed.ncbi.nlm.nih.gov/8639864
Publikováno v:
Cell Transplantation, Vol 4 (1995)
Treatment and potential cure of lysosomal and peroxisomal diseases, heretofore considered fatal, has become a reality during the past decade. Bone marrow transplantation, (BMT), has provided a method for replacement of the disease-causing enzyme defi
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::077a8027ae2aa4ad1c368909472c173f
https://doaj.org/article/5f66ae6f54704237801eecc377605b59
https://doaj.org/article/5f66ae6f54704237801eecc377605b59
Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation
Publikováno v:
Journal of inherited metabolic disease. 18(4)
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 characteri
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::653d0ecc13ef523ce4461b32656e7436
https://pubmed.ncbi.nlm.nih.gov/7494400
https://pubmed.ncbi.nlm.nih.gov/7494400