1288 results
1. To characterize long-term neurologic- and cognitive disorders, audiological and ophthalmological alterations in cCMV-infected children.2. To identify immunological, neurological host factors and virological factors that are predictive of…
The primary objective is to assess neuropsychological function in X-ALD patients without active cerebral demyelinating disease. Data of minors will be compared to matched healthy controls. Data of adults will be compared to reference values. The…
The aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with the study drug. The information gained from this study is expected to further characterize the efficacy and safety of BMN 044 over a…
Primary Objective:To evaluate the effect and sustainability over time of behavioural treatment for sleep problems in children with AS on the actual hours of sleep and sleep behaviour of the children.Secondary Objectives:To evaluate the effect and…
Specific AimsAs reviewed above, childhood anxiety disorders are highly prevalent and debilitating, and there is an urgent need for improvement of current treatment strategies. An innovative, but scarcely examined, treatment option for childhood…
The main objective of this study is to investigate whether a music intervention as complementary treatment is effective in reducing anxiety and distress around surgery. Secondary objectives are effects of music on pain, on preoperative anxiety, on…
Primary objective: - 50% seizure reduction or more at 3 months of dietary treatment .Secondary objectives: -level of ketosis on KD and MAD.-number of patients maintaining >50% seizure reduction on MAD.-number of withdrawals (not able to…
The purpose of this study is to assess the safety, tolerability and efficacy of long term drisapersen in subjects with DMD.
The primary objective is to obtain OCT data for children aged 4 to 10 diagnosed with non-syndromic craniosynostosis and to compare these to the normal reference values.
The aim of this project is to investigate the association between clinical and genetic factors and DDAVP response in MHA patients and to determine their relative contributions to the DDAVP response.
The main goal of the study is to evaluate the feasibility of using 1) passive treadmill belt speed perturbations to measure spinal reflex activity in the calf muscles; and 2) active real-time visual feedback on dedicated gait parameters to highlight…
Study i)Select the most valid and feasible accelerometer for measuring habitual physical activity in children with a mitochondrial disorderStudy ii)Measure the test-retest reliability of this instrument
The objective of this study is to obtain physiological data on lung aeration during pulmonary transition in newborn infants.
To evaluate the effects of long-term intranasal oxytocin on social behaviour in children with PWS and to compare the effects of different doses and frequencies of oxytocin administration
To evaluate the safety and tolerability of IV administration of SBC-103 in subjects with MPS lllB, Sanfilippo B.
To explore the safety, tolerability and the pharmacokinetic profile of 2-IB when given on top of therapeutic hypothermia.
To determine the accuracy of the URIKA urinary bladder sensor in measuring the distance between the anterior - and posterior wall of the bladder and to determine the range in bladder diameters at which the child should be alarmed to void.
PrimaryEffective treatment regimes and preventive tools of SIB in CdLS by studying phenomenology, etiology and pathogenesis.Secondary a. Development of a flow chart for daily care (diagnostics; management) b. Evaluation whether the present study can…
Primary objective:To assess the efficacy of BMN 045 after 48 weeks treatment in ambulant subjects with Duchenne muscular dystrophy.Secondary objectives:To assess the safety and tolerability of BMN 045 after 48 weeks of treatment in all study…
The current study is a continuation of the cohort 5-8 years post-diagnosis and aims to evaluate:1.The course of the nutritional status, and the related factors and consequences of malnutrition in survivors of childhood cancer until 5-8 years post-…