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Assessment and pharmacological correction of abnormalities in chloride (Cl-), bicarbonate (HCO3-) and mucus transport in rectal biopsies of CF patients

We aim to fill up the present gap in knowledge about HCO3- and mucus transport in native human intestine, and about possible effects of pharmacological correctors on these parameters in CF, by detailed electrophysiological and biochemical ex vivo…

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…

A phase IIb, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple subcutaneous doses of BMN 045 (previously known as PRO045) in subjects with Duchenne muscular dystrophy

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…

Postprandial bile acid metabolism in insulin sensitive and insulin resistant subjects:
The Bile Acid Study

To assess whether changes in insulin sensitivity through dietary interventions affect postprandial bile acid metabolism and kinetics. Secondary objective: to assess TGR5 and FXR mediated pathways in insulin resistant and insulin sensitive states.

Discrimination of benign and malignant human tissue during percutaneous interventions using optical spectroscopy techniques

In our previous studies using optical spectroscopy, both ex vivo and in vivo, most promising results were obtained for tissue diagnosis. Moreover, invasive procedures for biopsy or radiofrequency ablation in these tissues are all very common. Thus,…

Distinguishing children with frontal epilepsy from children with ADHD in terms the cognitive profile.

Distinguishing frontal lobe epilepsy from ADHD in terms of working memory.

A Long Term Follow-up Registry Study of Subjects Who Did Not Achieve Sustained Virologic Response in Gilead-Sponsored Trials in Subjects with Chronic Hepatitis C Infection

The primary objective of this Registry is:• To evaluate HCV viral sequences and the persistence or evolution of treatment emergent viral mutations in subjects who fail to achieve an SVR after treatment with a Gilead oral antiviral containing regimen…

Feasibility of MRI-imaging of the Affinis short shoulder prosthesis

To Establish the usefulness of MRI for assessment of the rotator cuff in patients with an Affinis short prosthesis implanted in the shoulder.

psychological consequences of pre-eclampsia

1. the primary objective of this study is to examen the incidence and prevalence of PTSDand depression in pre-eclampsia patients after delivery2. a secondary objective is to identify risk factors during pregnancy, durante partu and postpartum…

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.

An open label, long term, safety and tolerability extension to a randomized, double-blind, placebo controlled study of LCQ908 in subjects with Familial Chylomicronemia Syndrome

The purpose of this study is to determine long-term safety and tolerability, and continued efficacy in lowering triglycerides of LCQ908 in subjects FCS.

Cerebrovascular manifestations of Hereditary Hemorrhagic Telangiectasia: a look beyond AVMs | A study of the prevalence of structural brain changes in HHT and an assessment of the MR cerebrovascular reactivity of HHT patients

The objectives of this study are:1. to evaluate the occurrence and distribution of structural brain changes in HHT patients with and without pulmonary AVMs as compared to healthy age matched controls;2. to evaluate the (autoregulatory) function of…

Evaluation of the CAP-value for quantitating liver steatosis using 1H-MRS and liver biopsy as reference standard

Main objective: investigate the correlation between CAP-values with fat percentages found at 1H-MR Spectroscopy and steatosis grade at liver biopsy. Secondary objectives: investigate the reproducibility of CAP-measurements; investigate the…

Prove ART (Abluminal Reservoir Technology) clinical benefIt in *all comers* patients.

Evaluate clinical performances of Cre8 in *all comer* population

A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Biological Activity of SAR405838 in Patients with Advanced Cancer.

Primary objective* To determine safety and the maximum tolerated dose (MTD) of SAR405838 through the characterization of dose-limiting toxicities (DLTs).* To assess biological activities of SAR405838 in patients with dedifferentiated liposarcoma…

Facilitating the effective and early identification of children at risk for developing serious behavioral and emotional problems

In Phase 1 of the project, a systematic approach will be used to identify which factors add to the prediction of children*s clinical risk status. First, we will determine how well multiple assessments of behavioral problems (opposition, aggression,…

Associations between sleep bruxism and (peri-)implant complications: a follow-up study.

To investigate the time course in which (peri-)implant complications (i.e. inflammation and bone loss around dental implants and implant technical complications) develop and to identify the associations of sleep bruxism with these complications.

Leukocyte dynamics during immune reconstitution after stem-cell transplantation: in vivo labelling of dividing leukocytes using deuterated water

In this study we aim to gain more insights into immune reconstitution of various leukocyte populations after SCT. By application of heavy water labeling we investigate if and how the production- and death rate of leukocytes changes after an SCT.

Differentiated thyroid carcinoma in children: Late effects of treatment and pathophysiological background in the Netherlands

1) To study the late effects of 131-I treatment and TSH suppressive therapy as well as quality of life in patients with childhood-onset DTC. 2) To determine the presence of RET/PTC 1 and 3 translocations and BRAF mutations, and to investigate their…