320 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…
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…
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…
To assess the long-term safety, efficacy, and pharmacokinetics of oral lubiprostone 12 or 24 mcg capsules dosed twice daily (BID) when administered orally for 36 weeks in paediatric subjects with functional constipation. Evaluation of lubiprostone…
The aim of this study is to validate known, and identify novel allelic variants which may play a role in the risk of cisplatin-induced ototoxicity in a substantial international cohort of children diagnosed with cancer.
The main objective of this study is to identify rare, high-penetrance germline mutations predisposing to familial and/or early onset UBC.
18-22 YEAR OLD MALES WIHT A NORMAL WEIGHTExamine the possible attenuating cardiometabolic health effects of (A) hourly, 10-min standing interruptions, when compared to a single bout of prolonged sitting, and (B) sitting on a stability ball, when…
Research questions: Primary questions:* Are the new point-of-care instruments for the measurement of VOCs in exhaled breath feasible for use in children and adolescents aged 6 to 20 years?* Are these techniques able to differentiate between healthy…
Primary objective:To investigate the efficacy of a 12-week group-based training for female adolescents with CD (DBT-CD-A) compared to treatment as usual (TAU) within youth welfare institutions. It is hypothesised that add-on highly structured DBT-CD…
To study sensory processing and hyperexcitability in children with ASD and epilepsy using neurophysiological measurements (EEG/ERP).
The objective of the study is to investigate if it is possible to predict the clinical outcome of a patient with SCN1A related epilepsy based on the findings of advanced genotyping. We aim to answer the following questions:1. Can clinical outcome in…
The aim of this study is to investigate the effectiveness of a brief computer intervention to improve body satisfcation and self-esteem in a clinical population of adolescents with an eating disorder. We will examine whether the intervention is…