TY - JOUR
T1 - Mitochondrial dysfunction in Brooks-Wisniewski-Brown syndrome.
AU - Morava, Eva
AU - Rodenburg, R.
AU - Hol, F.
AU - De Meirleir, Linda
AU - Seneca, Sara
AU - Busch, R.
AU - Van Den Heuvel, L.
AU - Smeitink, Jan
PY - 2006/4
Y1 - 2006/4
N2 - Brooks, Wisniewski, and Brown described a familial presentation of severe developmental retardation, speech delay, static encephalopathy with atrophic hydrocephalus, microcephaly, progressive spastic diplegia, a characteristic facial appearance, optic atrophy, and growth retardation associated with hypoplastic corpus callosum in one of the patients. The authors postulated a distinct X-linked mental retardation syndrome. Later on a similar phenotype was observed in three male siblings with an early lethal outcome. Here we describe three patients with several overlapping features and a progressive neurological picture presenting with a significantly compromised mitochondrial oxidative phosphorylation measured in a fresh muscle biopsy. Neurological deterioration is a commonly observed feature in mitochondrial disorders. Based on the unique combination of the clinical symptoms, we suggest that our patients have the Brooks-Wisniewski-Brown syndrome.
AB - Brooks, Wisniewski, and Brown described a familial presentation of severe developmental retardation, speech delay, static encephalopathy with atrophic hydrocephalus, microcephaly, progressive spastic diplegia, a characteristic facial appearance, optic atrophy, and growth retardation associated with hypoplastic corpus callosum in one of the patients. The authors postulated a distinct X-linked mental retardation syndrome. Later on a similar phenotype was observed in three male siblings with an early lethal outcome. Here we describe three patients with several overlapping features and a progressive neurological picture presenting with a significantly compromised mitochondrial oxidative phosphorylation measured in a fresh muscle biopsy. Neurological deterioration is a commonly observed feature in mitochondrial disorders. Based on the unique combination of the clinical symptoms, we suggest that our patients have the Brooks-Wisniewski-Brown syndrome.
KW - Brooks-Wisniewski-Brown syndrome
M3 - Article
VL - 140
SP - 752
EP - 756
JO - American Journal of Medical Genetics Part A
JF - American Journal of Medical Genetics Part A
SN - 1552-4825
IS - April
ER -