Cerebellar Transcranial Direct Current Stimulation in Spinocerebellar Ataxia 38
Spinocerebellar AtaxiasSpinocerebellar ataxia 38 (SCA 38) is a very rare autosomal dominant inherited disorder caused by a mutation in ELOV5 gene, specifically expressed in cerebellar Purkinje cells, encoding an enzyme involved in the synthesis of fatty acids. The present study aimed to assess the effect of cerebellar anodal transcranial direct current stimulation (tDCS) administered employing deltoid (CD-tDCS) and spinal (CS-tDCS) cathodal montage. Clinical evaluation was performed at baseline (T0), after 15 sessions of tDCS (T1) and after one month of follow-up (T2).
Evaluation of the Effects of Calcitriol's in the Neurological Symptoms of Friedreich's Ataxia Patients...
Friedreich AtaxiaFriedreich's Ataxia (FA) is an autosomal recessive disease the mutation of which leads to a deficiency of a protein called frataxin, which is responsible for the symptoms of the disease. It is assumed that inducing an increase in the production of frataxin could reverse part of the disease's symptoms. Several treatments with drugs that raise frataxin levels have been tested, but they have either have not given the expected result or have induced intolerable side effects. The IRBLleida (Institut de Recerca Biomèdica de Lleida Fundació Dr. Pifarré) team has shown that calcitriol can increase the production of frataxin up to 2.5 to 3 times, a higher proportion than any of the drugs previously tested. For that reason, the next step in our research would be to check the effects of this drug (Calcitriol 0.25mcg/24h for a year) in patients with FA. On the other hand, calcitriol, the active form of vitamin D, is a drug with a very low rate of adverse effects that has been used for decades. Therefore, it is a drug with a very well established tolerability. The results of the present study, if positive, would lead to the organization of trials at a larger scale, and they would allow the use of an effective treatment for patients with FA.
Functional Trunk Training in Ataxia Patients
AtaxiaSpinocerebellar2 moreThe study is to examine the effect of functional trunk training on trunk control and upper extremity functions in patients with autosomal recessive ataxia.
Dual Task Training for Cerebellar Ataxia
Cerebellar AtaxiaCombining cognitive training with physical training to improve balance is a new approach for reducing the risk of falls in patient populations who are at risk for falls. People with brain pathology including cerebellar ataxia (CA) have difficulty in performing dual-tasks. Deficiency in dual-task performance relative to single-task performance referred to as dual-task cost is high in CA. Due to the high demands on cognitive resources, people with CA have higher falls rates during activities that involve dual tasking. Tai-Chi involves both cognition and physical movements making it a dual-tasking activity. However, previous study on the effects of 12-weeks of 8-form Tai-Chi did not demonstrate that it had beneficial effects in reducing falls among CA population. This null finding could potentially be due to (1) the lower levels of cognitive demands of Tai-Chi exercise, (2) the intervention not being intensive enough, or (3) the intervention may not have targeted the specific symptoms of CA. To determine if adding structured cognitive demands to conventional balance and coordination training (i.e., addressing all three possibilities for our previous null findings), the investigaotrs conducted a pilot study (n=5) to evaluate the feasibility, safety and benefits of a Cognitive-coupled Intensive Balance Training (CIBT) program. The more intensive and focused CIBT intervention reduced dual-task cost, improved balance, and reduced the number of falls in a sample of individuals with CA. Important next steps is to (1) evaluate the efficacy of the CIBT in a fully powered clinical trial, (2) understand the mechanisms underlying the benefits of CIBT training, and (3) determine the cost-benefits of this intervention. The hypothesis for the study includes (1) CIBT will improve balance and reduce falls; (2) reduction in dual-task cost of balance and cognitive performance will mediate a reduction in the number of falls in CA and (3) CIBT will be a cost-effective treatment option for improving balance and reduce falls. To test these hypotheses, a randomized controlled trial (RCT) with economic evaluation will be conducted over a period of two years to evaluate the effectiveness and cost-effectiveness of dual-task (CIBT) training compared with single-task (conventional balance: active control) training in individuals with CA.
Utilization of Motor Imagery Training for Improvement of Balance of Ataxic Children After Medulloblastoma...
Cerebellar AtaxiaBackground: after resection of medulloblastoma in children they suffer from signs and symptoms of ataxia which impedes their activities of daily living. purpose: to investigate the effect motor imagery training on balance, severity of ataxia and gait parameters on children after resection of medulloblastoma. Methods: Fifty children surfing from cerebellar ataxia after medulloblastoma resection were selected from tumors hospital of Cairo University, their age ranged from seven to nine years old, they were randomly assigned into two matched control and study groups. The control groups received the selected physical therapy program while, the study group received motor imaginary training in addition to the selected physical therapy program. Both groups were evaluated by ataxic rating scale, pediatric berg balance scale and kinematic gait analysis by kinovea software.
A Study of Resveratrol as Treatment for Friedreich Ataxia
Friedreich AtaxiaThe purpose of this study is to determine the effect of two doses of resveratrol taken for a 12 week period, on frataxin levels in individuals with Friedreich ataxia. This study will also measure the effect of resveratrol on markers of oxidative stress, clinical measures of ataxia, and cardiac parameters.
Effect of Pioglitazone Administered to Patients With Friedreich's Ataxia: Proof of Concept
Friedreich's AtaxiaFriedreich's ataxia (FA) is a rare progressive neurological disorder affecting approximately 1/30, 000 individuals. No treatment is presently available to counteract the neurodegeneration of this extremely severe disease. Pioglitazone, a well known PPAR gamma (peroxysome proliferators-activated receptor gamma) ligand induces the expression of many enzymes involved in the mitochondrial metabolism, including the superoxide dismutases. This agent may be therapeutic by counteracting the disabled recruitment of antioxidant enzymes in FA patients. This potential neuroprotective agent crosses the brain blood barrier in human. Primary objective: To explore the effects of Pioglitazone on neurological function in FA patients. We expect neurological benefits taking into account the natural course of the disease. Population: Subjects for this study will be limited to patients not older than 25 years Methodology: Prospective, randomized double-blind trial of Pioglitazone versus placebo in FA patients. Patients will be treated two years and will undergo clinical exams and testing during three days each six months at the clinical investigation centre.
A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia...
Friedreich's AtaxiaThe purpose of this trial is to study the efficacy, safety and tolerability of idebenone in 12 months of treatment in children and adults with Friedreich's Ataxia. This is a randomised placebo-controlled double-blind trial conducted in Europe. Efficacy outcomes include measures of neurological impairment and function, and measures of the heart.
Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's...
Friedreich's AtaxiaThis study is meant to assess the effectiveness of idebenone on neurological outcome measures in patients with Friedreich's Ataxia over a 6 months period.
Efficacy and Safety Study of Stemchymal® in Polyglutamine Spinocerebellar Ataxia
Cerebellar AtaxiaThe purpose of the clinical trial is to study the therapeutic efficacy and safety of Stemchymal® infusions for polyglutamine spinocerebellar ataxia treatment by a randomized, double-blind, placebo-controlled study design. Eligible subjects will receive Stemchymal® through intravenous infusion.