Long-term neurological outcome of a cohort of 80 patients with classical organic acidurias

被引:0
作者
Mathilde Nizon
Chris Ottolenghi
Vassili Valayannopoulos
Jean-Baptiste Arnoux
Valérie Barbier
Florence Habarou
Isabelle Desguerre
Nathalie Boddaert
Jean-Paul Bonnefont
Cécile Acquaviva
Jean-François Benoist
Daniel Rabier
Guy Touati
Pascale de Lonlay
机构
[1] APHP,Centre de Référence des Maladies Héréditaires du Métabolisme, Hôpital Necker
[2] Université Paris Descartes,Enfants Malades
[3] Institut Imagine,Service de Biochimie métabolique
[4] Hôpital Necker-Enfants Malades,Service de Neurologie, Hôpital Necker
[5] APHP,Enfants Malades
[6] Université Paris Descartes,Service de Radiologie Pédiatrique, Hôpital Necker
[7] APHP,Enfants Malades
[8] Université Paris Descartes,Service de Génétique Médicale, Hôpital Necker
[9] Institut Imagine,Enfants Malades
[10] APHP,Service de Maladies Héréditaires du Métabolisme
[11] Université Paris Descartes,Centre de Référence des Maladies Héréditaires du Métabolisme, Service de Biochimie
[12] Institut Imagine,undefined
[13] Centre de Biologie et Pathologie Est,undefined
[14] Hôpital Robert-Debré,undefined
[15] APHP,undefined
来源
Orphanet Journal of Rare Diseases | / 8卷
关键词
Organic aciduria; Propionic aciduria; Methylmalonic aciduria; Isovaleric aciduria; Neurological evolution; Long-term prognosis; Mitochondrial dysfunction;
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[1]  
Deodato F(2006)Methylmalonic and propionic aciduria Am J Med Genet C: Semin Med Genet 142C 104-112
[2]  
Boenzi S(2012)Clinical and neurocognitive outcome in symptomatic isovaleric acidemia Orphanet J Rare Dis 7 9-178
[3]  
Santorelli FM(2009)Long-term outcome in methylmalonic aciduria: a series of 30 French patients Mol Genet Metab 97 172-298
[4]  
Dionisi-Vici C(2006)Methylmalonic and propionic acidurias: management without or with a few supplements of specific amino acid mixture J Inherit Metab Dis 29 288-1029
[5]  
Grünert SC(2013)Propionic acidemia: clinical course and outcome in 55 pediatric and adolescent patients Orphanet J Rare Dis 8 6-423
[6]  
Wendel U(2012)Clinical variability of isovaleric acidemia in a genetically homogeneous population J Inherit Metab Dis 35 1021-389
[7]  
Lindner M(2005)Methylmalonic and propionic acidaemias: management and outcome J Inherit Metab Dis 28 415-639
[8]  
Leichsenring M(2006)“Classical” organic acidurias, propionic aciduria, methylmalonic aciduria and isovaleric aciduria: long-term outcome and effects of expanded newborn screening using tandem mass spectrometry J Inherit Metab Dis 29 383-110
[9]  
Schwab KO(2009)Prediction of outcome in isolated methylmalonic acidurias: combined use of clinical and biochemical parameters J Inherit Metab Dis 32 630-37
[10]  
Vockley J(2013)Renal transplantation in 4 patients with methylmalonic aciduria: A cell therapy for metabolic disease Mol Genet Metab 110 106-67