Distinct neurological disorders with ATP1A3 mutations

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Distinct neurological disorders with ATP1A3 mutations.

Genetic research has shown that mutations that modify the protein-coding sequence of ATP1A3, the gene encoding the α3 subunit of Na(+)/K(+)-ATPase, cause both rapid-onset dystonia parkinsonism and alternating hemiplegia of childhood. These discoveries link two clinically distinct neurological diseases to the same gene, however, ATP1A3 mutations are, with one exception, disease-specific. Althoug...

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Intermediate Phenotypes of ATP1A3 Mutations: Phenotype–Genotype Correlations

BACKGROUND ATP1A3-related disorders include rapid-onset dystonia-parkinsonism (RDP or DYT12), alternating hemiplegia of childhood (AHC), and CAPOS syndrome (Cerebellar ataxia, Areflexia, Pes cavus, Optic atrophy, and Sensorineural hearing loss). CASE REPORT We report two cases with intermediate forms between RDP and AHC. Patient 1 initially presented with the AHC phenotype, but the RDP phenot...

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Unravelling the pathophysiology of calcium channel mutations causing neurological disorders.

Voltage-gated calcium channels are molecules that play a evoked release of acetylcholine at the neuromuscular junction (Plomp et al., 2000). They show that high-rate stimulation key role in cell-to-cell communication and muscle contracleads to an increased run-down of evoked acetylcholine tion. They are divided into low voltage-activated (LVA or Trelease in homozygous tg mouse compared with the...

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Neurological disorders caused by inherited ion-channel mutations.

Several neurological diseases-including neuromuscular disorders, movement disorders, migraine, and epilepsy-are caused by inherited mutations of ion channels. The list of these "channelopathies" is expanding rapidly, as is the phenotypic range associated with each channel. At present the best understood channelopathies are those that affect muscle-fibre excitability. These channelopathies produ...

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Sodium channel mutations in epilepsy and other neurological disorders.

Since the first mutations of the neuronal sodium channel SCN1A were identified 5 years ago, more than 150 mutations have been described in patients with epilepsy. Many are sporadic mutations and cause loss of function, which demonstrates haploinsufficiency of SCN1A. Mutations resulting in persistent sodium current are also common. Coding variants of SCN2A, SCN8A, and SCN9A have also been identi...

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ژورنال

عنوان ژورنال: The Lancet Neurology

سال: 2014

ISSN: 1474-4422

DOI: 10.1016/s1474-4422(14)70011-0