TY - JOUR
T1 - Therapeutic Advances in Neurodegeneration With Brain Iron Accumulation
AU - Zorzi, Giovanna
AU - Zibordi, Federica
AU - Chiapparini, Luisa
AU - Nardocci, Nardo
PY - 2012/6
Y1 - 2012/6
N2 - Neurodegeneration with brain iron accumulation (NBIA) includes a heterogeneous group of genetically defined disorders characterized by progressive extrapyramidal deterioration and iron accumulation in the basal ganglia. Current medical options for these disorders remain largely unsatisfactory and do not prevent the disease from progressing to a severe and disabling state. In select cases, surgical techniques, such as deep brain stimulation, may be effective in ameliorating some of the symptoms of the disease. The availability of chelating agents with specific properties that have been demonstrated to be effective in other disorders with regional iron accumulation as well as magnetic resonance imaging techniques that allow for quantitative assessment of iron have stimulated interest in the use of chelating agents in NBIA. This review aims to describe the role of surgical therapies in NBIA, discuss the use of chelating agents in NBIA, and presents new therapeutic approaches under consideration.
AB - Neurodegeneration with brain iron accumulation (NBIA) includes a heterogeneous group of genetically defined disorders characterized by progressive extrapyramidal deterioration and iron accumulation in the basal ganglia. Current medical options for these disorders remain largely unsatisfactory and do not prevent the disease from progressing to a severe and disabling state. In select cases, surgical techniques, such as deep brain stimulation, may be effective in ameliorating some of the symptoms of the disease. The availability of chelating agents with specific properties that have been demonstrated to be effective in other disorders with regional iron accumulation as well as magnetic resonance imaging techniques that allow for quantitative assessment of iron have stimulated interest in the use of chelating agents in NBIA. This review aims to describe the role of surgical therapies in NBIA, discuss the use of chelating agents in NBIA, and presents new therapeutic approaches under consideration.
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U2 - 10.1016/j.spen.2012.03.007
DO - 10.1016/j.spen.2012.03.007
M3 - Article
C2 - 22704261
AN - SCOPUS:84862319391
VL - 19
SP - 82
EP - 86
JO - Seminars in Pediatric Neurology
JF - Seminars in Pediatric Neurology
SN - 1071-9091
IS - 2
ER -