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The effect of oxytocin administration on empathy and emotion recognition in healthy and antisocial adolescents

Our primary objective is to investigate whether oxytocin administration can affect empathy and emotion recognition in healthy and antisocial adolescents. We expect that oxytocin will enhance empathy and emotion recognition in both healthy and…

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…

Web-based attention bias modification treatment for childhood anxiety disorders: a randomized, double-blind, controlled trial

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…

A placebo-controlled discontinuation trial of off-label used risperidone in people with intellectual disability

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…

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.

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.

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-…

Treatment of severe acute GVHD after allogeneic hematopoietic stem cell transplantation with steroids versus MSC and steroids.
A prospective double-blind placebo-controlled randomized phase III trial

To improve the response rate to treatment of severe acute GVHD (grade II-IV with gut involvement ) by adding infusion of Mesenchymal Stroma Cells to standard high dose prednisolone.

Scar quality in Burn Wounds with Various Healing Potentials:A Pilot Study

To determine long-term scar quality in patients admitted to a Dutch burn centre with burn wound of indeterminate depth.

Proactive versus reactive inhibition in ADHD: Genetics, Neurobiology and pharmacology

The aim of this research is, following the aforementioned theoretical framework, to investigate whether the two candidate genes in combination with EEG and fMRI markers differentiate between children that do and do not show a clinical response to…

Improvement of Diagnostic mEthods for ALlergy assessment. Cashew allergy in children as a showcase (IDEAL study)

To improve diagnostic tools for food/cashew nut allergies with the use of Mediator Release Assays.Genetic component of cashewnut allergy.

Unrelated cord blood transplantation after reduced toxicity conditioning with mesenchymal stromal cell co-infusion in patients with severe epidermolysis bullosa

We propose a phase II study (intervention) in patients with severe generalized recessive dystrophic EB receiving reduced toxicity conditioning chemotherapy followe by cord blood transplantation with co-infusion of mesenchymal stromal cell units.…

Determination whether the lack of functional AQP2 in kidneys of patients suffering from nephrogenic diabetes insipidus induces cyst formation

Does the lack of the Aquaporin-2 water channel cause cyst formation in patients suffering from nephrogenic diabetes insipidus?