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Year Number of Results
1970 1
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1989 1
1991 2
1993 3
1994 3
1995 2
1997 2
1998 3
1999 7
2000 6
2001 2
2002 4
2003 8
2004 2
2005 8
2006 7
2007 4
2008 1
2009 1
2010 2
2011 2
2012 6
2013 5
2014 4
2015 2
2016 4
2017 2
2018 1
2019 1
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2022 1
2023 2

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98 results

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Page 1
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, Gray SJ, Kay CN, Boye SL, Boye SE, George LA, Salabarria S, Corti M, Byrne BJ, Tremblay JP. Mendell JR, et al. Mol Ther. 2021 Feb 3;29(2):464-488. doi: 10.1016/j.ymthe.2020.12.007. Epub 2020 Dec 10. Mol Ther. 2021. PMID: 33309881 Free PMC article. Review.
We review gene therapy for neuromuscular disorders (spinal muscular atrophy [SMA]; Duchenne muscular dystrophy [DMD]; X-linked myotubular myopathy [XLMTM]; and diseases of the central nervous system, including Alzheimer's disease, Parkinson's diseas …
We review gene therapy for neuromuscular disorders (spinal muscular atrophy [SMA]; Duchenne muscular dystrophy [DMD]; X-linked myotubular my …
Mycoplasma pneumoniae-induced rash and mucositis as a syndrome distinct from Stevens-Johnson syndrome and erythema multiforme: a systematic review.
Canavan TN, Mathes EF, Frieden I, Shinkai K. Canavan TN, et al. J Am Acad Dermatol. 2015 Feb;72(2):239-45. doi: 10.1016/j.jaad.2014.06.026. J Am Acad Dermatol. 2015. PMID: 25592340 Review.
OBJECTIVE: We sought to review the literature characterizing morphology and disease course of M pneumoniae-associated mucocutaneous disease. METHODS: A comprehensive literature search identified 95 articles with 202 cases. ...Because of the distinct morphology, mild …
OBJECTIVE: We sought to review the literature characterizing morphology and disease course of M pneumoniae-associated mucocutaneous …
Leukodystrophies.
Perlman SJ, Mar S. Perlman SJ, et al. Adv Exp Med Biol. 2012;724:154-71. doi: 10.1007/978-1-4614-0653-2_13. Adv Exp Med Biol. 2012. PMID: 22411242 Review.
The clinical characteristics, etiologies, diagnostic testing and treatment options are reviewed in detail for some of the major leukodystrophies: X-linked adrenoleukodystrophy, Krabbe disease, metachromatic leukodystrophy, Pelizaeus-Merzbacher disease, Alexander …
The clinical characteristics, etiologies, diagnostic testing and treatment options are reviewed in detail for some of the major leukodystrop …
Cervical cancer.
Canavan TP, Doshi NR. Canavan TP, et al. Am Fam Physician. 2000 Mar 1;61(5):1369-76. Am Fam Physician. 2000. PMID: 10735343 Free article. Review.
Accurate and early recognition of abnormal cytologic changes prevents progression of the disease from preinvasive to invasive. Research is under way to determine if efforts to reduce the false-negative rate of the Papanicolaou smear should include rescreening programs and …
Accurate and early recognition of abnormal cytologic changes prevents progression of the disease from preinvasive to invasive. Resear …
Merkel cell carcinoma.
Han SY, North JP, Canavan T, Kim N, Yu SS. Han SY, et al. Hematol Oncol Clin North Am. 2012 Dec;26(6):1351-74. doi: 10.1016/j.hoc.2012.08.007. Epub 2012 Oct 9. Hematol Oncol Clin North Am. 2012. PMID: 23116583 Review.
Surgery and/or radiation is of the mainstay of therapy for early disease, while chemotherapy is reserved for more advanced disease. ...
Surgery and/or radiation is of the mainstay of therapy for early disease, while chemotherapy is reserved for more advanced disease …
Non-genetic therapeutic approaches to Canavan disease.
Roscoe RB, Elliott C, Zarros A, Baillie GS. Roscoe RB, et al. J Neurol Sci. 2016 Jul 15;366:116-124. doi: 10.1016/j.jns.2016.05.012. Epub 2016 May 10. J Neurol Sci. 2016. PMID: 27288788 Review.
Canavan disease (CD) is a rare leukodystrophy characterized by diffuse spongiform white matter degeneration, dysmyelination and intramyelinic oedema with consequent impairment of psychomotor development and early death. ...The physiological role of ASPA is to …
Canavan disease (CD) is a rare leukodystrophy characterized by diffuse spongiform white matter degeneration, dysmyelina …
Selected leukodystrophies.
Powers JM, Rubio A. Powers JM, et al. Semin Pediatr Neurol. 1995 Sep;2(3):200-10. doi: 10.1016/s1071-9091(05)80031-2. Semin Pediatr Neurol. 1995. PMID: 9422247 Free article. Review.
The clinicopathologic profiles of the major leukodystrophies (adreno-leukodystrophy, metachromatic leukodystrophy, globoid cell leukodystrophy or Krabbe's disease, Pelizaeus-Merzbacher disease, and spongy degeneration of infancy or Canavan's …
The clinicopathologic profiles of the major leukodystrophies (adreno-leukodystrophy, metachromatic leukodystrophy, globoid cell leukodystrop …
Spongy degeneration of the brain, Canavan disease: biochemical and molecular findings.
Matalon RM, Michals-Matalon K. Matalon RM, et al. Front Biosci. 2000 Mar 1;5:D307-11. doi: 10.2741/matalon. Front Biosci. 2000. PMID: 10704428 Review.
Canavan disease is a severe progressive leukodystrophy characterized by swelling and spongy degeneration of the white matter of the brain. ...Hypotonia, head lag and macrocephaly are characteristic of Canavan disease and become apparent a …
Canavan disease is a severe progressive leukodystrophy characterized by swelling and spongy degeneration of the …
Recent Advancements in the Diagnosis and Treatment of Leukodystrophies.
Bradbury AM, Ream MA. Bradbury AM, et al. Semin Pediatr Neurol. 2021 Apr;37:100876. doi: 10.1016/j.spen.2021.100876. Epub 2021 Feb 10. Semin Pediatr Neurol. 2021. PMID: 33892849 Review.
Numerous preclinical studies have shown safety and efficacy of adeno-associated virus gene therapy in leukodystrophies allowing expanded access treatment for Canavan disease prior to initiation of a clinical trial. For inherited white matter disorders resulting from …
Numerous preclinical studies have shown safety and efficacy of adeno-associated virus gene therapy in leukodystrophies allowing expanded acc …
Leukodystrophies with astrocytic dysfunction.
Rodriguez D. Rodriguez D. Handb Clin Neurol. 2013;113:1619-28. doi: 10.1016/B978-0-444-59565-2.00030-7. Handb Clin Neurol. 2013. PMID: 23622383 Review.
Astrocytic dysfunctions have been recently identified in four leukosdystrophies without peripheral nervous system myelin involvement. Alexander disease, the first primary genetic astrocytic disorder identified, is due to dominant GFAP mutations. ...An increas …
Astrocytic dysfunctions have been recently identified in four leukosdystrophies without peripheral nervous system myelin invol …
98 results