March 2011
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13 Reads
Neuropediatrics
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March 2011
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13 Reads
Neuropediatrics
November 2010
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78 Reads
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63 Citations
The Lancet Neurology
Background: Duchenne muscular dystrophy is a rare X-linked progressive disease characterised by loss of ambulation at about age 10 years, with death in early adulthood due to respiratory and cardiac insufficiency. Steroids are effective at slowing the progression of muscle weakness; however, their use is limited by side-effects, prompting the search for alternatives. We assessed the effect of ciclosporin A as monotherapy and in combination with intermittent prednisone for the treatment of ambulant patients with this disorder. Methods: Our study was a parallel-group, placebo-controlled, double-blind, multicentre trial at trial sites of the German muscular dystrophy network, MD-NET, over 36 months. Ambulant patients with Duchenne muscular dystrophy who were aged 5 years or older were randomly assigned to receive either ciclosporin A (3·5-4·0 mg/kg per day) or matching placebo. Allocation was done centrally with computer-generated random numbers. Patients and investigators were masked to the allocated treatment. After 3 months of treatment, both groups were also given intermittent prednisone for a further 12 months (0·75 mg/kg, alternating 10 days on with 10 days off). All patients who received at least one dose of study drug or placebo were included in the primary analysis. The primary outcome measure was manual muscle strength measured on the Medical Research Council (MRC) scale. This trial is registered with the German clinical trial register DRKS, number DRKS00000445. Findings: 77 patients were randomly assigned to the ciclosporin A group and 76 to the placebo group; 73 patients on ciclosporin A and 73 on placebo received at least one dose and were available for efficacy analyses. 3 months of treatment with ciclosporin A alone did not show any significant improvement in primary outcome measures (mean change in the proportion of a possible total MRC score [%MRC] was -2·6 [SD 6·0] for patients on ciclosporin A and -0·8 [4·9] for patients on placebo; adjusted group difference estimate -0·88, 97·5% CI -2·6 to 0·9; p=0·26). The combination of ciclosporin A with intermittent steroids was not better than intermittent steroids alone over 12 months (mean change in %MRC was 0·7 [7·1] for patients on ciclosporin A and -0·3 [7·9] for patients on placebo; adjusted group difference estimate -0·85, -3·6 to 1·9; p=0·48). Numbers of adverse events (75 in patients on ciclosporin A and 74 on placebo) and serious adverse events (four with ciclosporin A and four with placebo) did not differ significantly between groups. Interpretation: Ciclosporin A alone or in combination with intermittent prednisone does not improve muscle strength or functional abilities in ambulant boys with Duchenne muscular dystrophy, but is safe and well tolerated. Funding: German Federal Ministry of Education and Research, Action Benni and co eV, Novartis Pharma AG, and Deutsche Gesellschaft für Muskelkranke eV.
October 2008
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94 Reads
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12 Citations
Brain and Development
We report our normative data of somatosensory evoked potentials (SEP) after posterior tibial nerve (PTN) stimulation from a group of 89 children and 18 adults, 0.4-29.2 years of age. We recorded near-field potentials from the peripheral nerve, the cauda equina, the lumbar spinal cord and the somatosensory cortex. Far-field potentials were recorded from the scalp electrodes with a reference at the ipsilateral ear. N8 (peripheral nerve) and P40 (cortex) were present in all children but one. N20 (cauda equina) and N22 (lumbar spinal cord) were recorded in 94 and 106 subjects, respectively. P30 and N33 (both waveforms probably generated in the brainstem) were recorded in 103 and 101 subjects, respectively. Latencies increased with age, while central conduction times including the cortical component, decreased with age (up to about age 10 years). The amplitudes of all components were very variable in each age group. We report our normative data of the interpeak latencies N8-N22 (peripheral conduction time), N22-P30 (spinal conduction time), N22-P40 (central conduction time) and P30-P40 (intracranial conduction time). These interpeak latencies should be useful to assess particular parts of the pathway. The subcortical PTN-SEPs might be of particular interest in young or retarded children and during intraoperative monitoring, when the cortical peaks are influenced by sedation and sleep, or by anesthesia.
January 2007
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58 Reads
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7 Citations
Manuelle Medizin
January 2007
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285 Reads
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7 Citations
Manuelle Medizin
Purpose To assess the therapeutic efficacy of osteopathic treatment in infants with postural asymmetry. Patients and methods A randomized clinical trial of efficacy with blinded videoscoring was performed. Sixty-one infants with postural asymmetry aged 6–12 weeks (median: 9) were recruited. Thirty-two infants (18 male, 14 female) with a gestational age ≥36 weeks were found to be eligible and randomly assigned to the intervention groups; 16 received osteopathic treatment and 16 sham therapy. After a treatment period of 4 weeks the outcome was measured using a standardised scale (40–24 points). Results With sham therapy, five infants improved (=3 points), eight infants were unchanged (±<3 points) and three infants deteriorated (>−3 points); the mean improvement was 1.2 points (SD±3.5). In the osteopathic group 13 infants improved and 3 remained unchanged; the mean improvement was 5.9 points (SD±3.8). The difference was significant (p=0.001). Conclusion Osteopathic treatment in the first months of life improves the degree of asymmetry in infants with postural asymmetry.
December 2006
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4 Reads
Neuropediatrics
September 2006
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16 Reads
Neuropediatrics
February 2006
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357 Reads
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97 Citations
The aim of this study was to assess the therapeutic efficacy of osteopathic treatment in infants with postural asymmetry. A randomized clinical trial of efficacy with blinded videoscoring was performed. Sixty-one infants with postural asymmetry aged 6 to 12 weeks (mean 9wks) were recruited. Thirty-two infants (18 males, 14 females) with a gestational age of at least 36 weeks were found to be eligible and randomly assigned to the intervention groups, 16 receiving osteopathic treatment and 16 sham therapy. After a treatment period of 4 weeks the outcome was measured using a standardized scale (4-24 points). With sham therapy, five infants improved (at least 3 points), eight infants were unchanged (within 3 points), and three infants deteriorated (not more than -3 points); the mean improvement was 1.2 points (SD 3.5). In the osteopathic group, 13 infants improved and three remained unchanged; the mean improvement was 5.9 points (SD 3.8). The difference was significant (p=0.001). We conclude that osteopathic treatment in the first months of life improves the degree of asymmetry in infants with postural asymmetry.
April 2005
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7 Reads
Neuropediatrics
September 2003
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16 Reads
Klinische Neurophysiologie
... В проект новой классификации эпилептических синдромов Международной противоэпилептической лиги введена рубрика эпилептических энцефалопатий [1]. Она включает хронические расстройства, при которых главную проблему составляют тяжелые мозговые дисфункции, обусловленные эпилептическими разрядами, проявляющимися в ЭЭГ как эпилептиформная активность [1][2][3][4][5][6][7][8][9]. В эту рубрику внесены синдромы и формы известных ранее «катастрофических эпилептических энцефалопатий», при которых частые и тяжелые эпилептические припадки сочетаются с тяжелой задержкой или регрессом психоневрологического развития: синдромы Отахара, Уэста, Леннокса-Гасто, Драве и др. ...
January 1999
Epilepsia
... Zeigt sich hierunter keine Abnahme der Haltungsasymmetrie, sollte zeitnah eine erweiterte Diagnostik stattfi nden. OMT (ebenso wie Vojta-oder Bobath-Therapie) ist hier als nicht-invasiv und nebenwirkungsarm zu sehen und bietet so einen schnellen und effi zienten sowie kostengünstigen Ansatz bei infantiler Haltungsasymmetrie mit lagebedingter Plagiozephalie[20],[21]. Bei einer Therapieresistenz mit persistierender Haltungsasymmetrie trotz Verbesserung der funktionellen Befunde ist die Kommunikation mit dem behandelnden Pädiater aufzunehmen. ...
January 2007
Manuelle Medizin
... The patients typically manifest hepatosplenomegaly and progressive neurological symptoms including vertical supranuclear ophthalmoplegia, progressive ataxia, dystonia, and dementia, with varying age at onset. Interestingly, several cases of NPC with cataplexy have been reported (Kandt et al. 1982;Denoix et al. 1991;Challamel et al. 1994;Boor and Reitter 1997). In cataplexy, sudden and brief episodes of bilateral loss of muscle tone are observed during strong emotions such as laughing or anger. ...
March 1997
Klinische Pädiatrie
... The potential applications of OMT to pediatrics are many, and extend to areas other than musculoskeletal problems [25][26][27]. Despite a number of studies suggesting a benefit of OMT in conditions such as sucking difficulties [28], colic [29], dacryostenosis [30], plagiocephaly [31][32][33], torticollis [34], otitis media [35,36], voiding difficulties [37], scoliosis [38,39], headaches [40][41][42], concussions [43,44], and more chronic conditions [45] such as cerebral palsy [46] and attention deficit disorder (ADD) [47], studies are often challenged because of their methodology. ...
January 2007
Manuelle Medizin
... Consistently, preventing PTP opening by cyclosporin A (CsA) or its analog alisporivir, both targeting cyclophilin D whose activity favors PTP opening, improves the mdx phenotype, and restores mitochondria activity. However, CsA treatment fails to improve muscle function in DMD patients [48] and exhibits extensive immunosuppressive effects. Even though alisporivir normalizes mitochondria calcium retention and respiration, it suppresses mitochondrial biogenesis, organelle dynamics, and mitophagy in both cardiac and skeletal muscle [49,50]. ...
November 2010
The Lancet Neurology
... As patients with mitochondrial disease frequently demonstrate variable CNS involvement (Loeffen et al. 2000, Kim et al. 2008, they are at risk of having posterior visual pathway damage and visual perceptual problems. Optic nerve hypoplasia (ONH) has also been described in relation to mitochondrial disease (Boor et al. 1992, Taban et al. 2006. ...
August 1992
European Journal of Pediatrics
... For this reason, an electroretinogram (ERG) is also one of the electrophysiological studies [139,165]. It is a tool that allows for the monitoring of retinal involvement, and its use gradually disappears as the disease progresses, depending on the variants of NCL [165,175]. The use of ERG might be implemented in the future to evaluate the efficacy of experimental treatments with intravitreal therapies [165]. ...
March 1995
Brain and Development
... There have been suggestions that surgery should be undertaken if the Cobb angle is between 20 and 50°567 . Early operation has been shown to improve outcome in long term follow up studies [3, 4, 8, 9]. Surgical techniques described so far in scoliosis correction in DMD patients range from halo casts with traction wires and buttons [10], Harrington rods [11], Luque's segmental spinal fixation [12] to more recent techniques using pedicle screws and hooks. ...
January 1994
Zeitschrift für Orthopädie
... SEP is the recording of timelocked responses to external electrical stimulation of peripheral nerves, which can be recorded at various neural levels such as the spinal cord or cortex. 17 Duckworth et al. showed that SEP findings were well correlated with clinical findings in patients with spina bifida in their study. SEP was shown to be an indicator of the integrity of neural pathways between the point of electrical stimuli (S1 dermatome) and cerebral cortex (somatosensory cortex), and the absence of electrical signal transmission was considered a neural injury. ...
October 1993
Child's Nervous System
... 116 Dexamethasone is rarely mentioned as an add-on treatment for neonatal seizures; however, some authors do recommend it in their algorithms. 117,118 The dosage is 0.6-2.8 mg/kg intravenously and is usually divided into four daily doses given for 3-10 consecutive days 118 (see Table 2). ...
February 1993
Acta Paediatrica