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Neuropsychological assessment in X-linked adrenoleukodystrophy

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…

Think different. A study into the feasibility and the effectiveness of an online Cognitive Bias Modification training on an app for adolescents with an obsessive compulsive disorder (OCD)

This study concerns research into the applicability and effectiveness of CBM-I training through an app on the mobile phone. The research is done in adolescents (12-18 years) treated at the Bascule clinic for OCD. The training will take place during…

EMDR with traumatized adolescents with a major depressive disorder

The aim of this study is to investigate the effectiveness of EMDR with traumatized adolescents (12-18 years) with major depressive disorder.

Inguinal or Ilio-inguinal Lymphadenectomy for Patients With Metastatic Melanoma to Groin Lymph Nodes and no Evidence of Pelvic Disease on PET/CT Scan - A Randomised Phase III Trial (EAGLE FM)

This study aims to provide a more rational evidence base for appropriate management for metastatic melanoma in the groin LNs, through assessing the effect of the addition of ipsilateral pelvic lymphadenectomy on patient disease-free survival (DFS),…

A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy

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…

An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy.

The purpose of this study is to assess the safety, tolerability and efficacy of long term drisapersen in subjects with DMD.

Paediatric distal radius torus fracture treatment comparison in a prospective randomized control trial: mitella versus plaster cast

If the study shows that the treatment with a sling gives an earlier return to normal function en does not give a clinically significant difference in pain experience compared to cast therapy than the traditional treatment can be adjusted. We aim at…

Response to DDAVP In mild Hemophilia A patients, in Search for dEterminants

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.

A multicenter randomized controlled trial comparing long term scar quality after hydrosurgical versus conventional tangential excision of deep dermal burns

To assess and compare long term scar quality of deep dermal burns after debridement with hydrosurgical- and conventional tangential excision.

Gait Analysis in an iMmersive Enviroment

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…

Four-dimensional flow cardiac magnetic resonance imaging for the assessment of coarctation of the aorta, a monocenter, prospective, diagnostic cross-sectional study.

1. To investigate non-invasive 4D flow CMR for the assessment of aortic arch stenosis and compare and correlate this to conventional imaging modalities and to three-dimensional rotational angiography (3DRA) with computational fluid dynamics (CFD). 2…

Validity and feasibility of accelerometers for assessing habitual physical activity in children with mitochondrial disease.

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

Intranasal administration of oxytocin in children with Prader-Willi Syndrome. A randomized, open-label, cross-over trial of different treatment regimens of oxytocin administration. Effects on eating behaviour and social behaviour.

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

A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Biodistribution, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

To evaluate the safety and tolerability of IV administration of SBC-103 in subjects with MPS lllB, Sanfilippo B.

National observational study to monitor the new guideline concerning treatment of atypical hemolytic uremic syndrome

Monitoring and evaluation of the Dutch guideline for treatment of aHUS in children and adults during two years.

CoDeLaGe
A translational cohort study on self-injurious behaviour in people with Cornelia de Lange Syndrome

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…

Physical and psychological sequelae of childhood cancer from diagnosis till 5-8 years after diagnosis

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-…

Differentiating simple from complex appendicitis in children before surgery: The Complex Appendicitis Tool (CAT)

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…

APC & Behaviour

The above anounced studies suggest that some abnormalities in the Wnt pathway can be expected to affect development and function of the brain, potentially leading to recognizable behavioural phenotypes. Given the key role of APC in the Wnt pathway,…

Prospective comparative study between Deflux® and Vantris® for endoscopic treatment of vesico-ureteral reflux in children.

Primary Objective: The treatment result expressed as the proportion of successful treatments per treatment arm (successful = VUR <= grade 1 and/or no urinary-tract infections 6 months after the operation) does at least not show a significant…