319 results
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…
The primary objective of this study is: To study the effect of controlled discontinuation of long-term used risperidone, for the treatment of challenging behavior, on behaviour and health. Our hypothesis is that long-term use of risperidone for…
The purpose of this study is to assess the safety, tolerability and efficacy of long term drisapersen in subjects with DMD.
To evaluate the safety and tolerability of IV administration of SBC-103 in subjects with MPS lllB, Sanfilippo B.
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-…
1. Immunology and Pathology:To determine the immune response of patients with simple and complex appendicitis in order to identify markers which distinguish between simple and complex appendicitis.2.Clinical and Imaging:To analyze the differences in…
Main question: What is the lung function of late preterm births compared with children born at term at age 12? Sub-questions: - Which respiratory symptoms in late preterm infants at age 12?
Main: 1. To investigate whether a short term CBT-based group program is more effective in reducing symptoms of depression and anxiety than treatment as usual (TAU), among adolescents aged 10-16 within residential treatment settings in the youth…
To determine long-term scar quality in patients admitted to a Dutch burn centre with burn wound of indeterminate depth.
Objective: To test the hypotheses that Feedback Informed Treatment together with treatment as usual will show significant more improvement than treatment as usual alone.
This study aims to compare the effects of a training in concrete thinking and a training in positive reappraisal with a control conditions (no specific training instructions) on indices of psychological wellbeing in youth aged 15 to 18. Participants…
1. To identify genetic variants associated with an increased risk of ASD using different genetic models (dominant, recessive, interaction).2. To determine the relationship between different genetic variants and the associated neuropsychiatric ASD…
(i) identification and cellular characteristics of red blood cells from patients with altered ion homeostasis and delivery of novel and easy-to-use diagnostics for rare anemia*s, for patients with: a) a known primary defect in the ion channels of…
Primary objective is to objectively investigate the therapeutic potential of Ivacaftor in Dutch CF patients carrying a mutation associated with residual CFTR function. A secondary objective is to evaluate the correlations between individual…
1) To perform gene mutation analysis in family members of patients carrying a proven mutation in the candidate gene.2) To determine the clinical, biochemical and radiological characteristics of carriers of a mutation in the candidate gene.3) To…
(1) To determine the frequency of antibody-mediated encephalopathy in children with seizure-related or (sub)acute onset neuropsychiatric disorders. (2) To identify the target auto-antigens of selected seizure-related and (sub)acute onset…
Main objective: To investigate Differences in sustainability of inhalation technique of Diskus® and Autohaler® in the home situation. This will be measured with the inhalation checklist for the Diskus® and Autohaler®. Secondary objectives: * To…
The objective of this study is to evaluate whether an in- or outpatient initiation of the brace treatment has an influences short and long term brace compliance.
The primary objective is to assess the treatment effect on response rate (MDS: either completeremission [CR], partial remission [PR], or marrow CR; JMML: either clinical completeremission [cCR] or clinical partial remission [cPR]); at Cycle 3 Day 28…
The primary objective of the current study is to (further) investigate the role of reward and punishment sensitivity in Anorexia Nervosa and obesity by examining general and disorder-specific appetitive and aversive biases. The secondary objective…