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"Metabolomics from*lysosomal organelles*isolated*from fresh peripheral blood of CLN3 patients and healthy controls and/or allele carriers".*A proof of principle study.

We aim to define components which are critical for lysosomal homeostasis and are affected in CLN3 disease. To this end we will perform multi-omics analysis of lysosomes isolated from fresh peripheral white blood mononuclear cells (PBMCs) of…

The Effectiveness of Virtual Reality Exposure Therapy in Adolescents with Social Anxiety - a Single Case Experimental Design

The primary goal of this study is to evaluate the effectiveness of VR-exposure therapy (VRET) in diminishing anxiety and avoidance within adolescents with social anxiety. We expect that, compared to the within-person baseline and standard treatment…

Facing fears together - a pilot study on peer-mentored cognitive behavioral therapy for adolescents with mild intellectual disability and anxiety

Primary Objective: Is the intervention model feasible and acceptable for clients, parents/caregivers, peers and therapists?Secondary Objective(s): 1) Establishing first evidence of efficacy of peer-mentored CBT in reducing anxiety in adolescents…

Children's Health Assessment and Molecular Pathogen Identification for Optimized Novel Sepsis therapy

The aim of this study is to establish the accuracy of Molecular Culture for prediciting the outcome of the traditional blood culture.

Spinal Muscular Atrophy Randomized Controlled Trial on Cough Techniques
(Patient tailored airway clearance in patients with Spinal Muscular Atrophy: a randomized controlled trial comparing air stacking and mechanical insufflation-exsufflation.)

The aim of this study is to compare the effect of twice daily MI-E to twice daily AS with MAC on increase of PECF in patients with SMA with weak cough over a period of 3 years.

Utilization of the Navigate Anti-AAV9 Antibody Assay in Support of the Novartis* Clinical Studies: COAV101B12301 *STEER* and COAV101B12302 *STRENGTH*

The primary objective of this study is to evaluate the performance of the Navigate Anti-AAV9 Antibody Assay using serum specimens in subjects with SMA in Novartis clinical studies COAV101B12301 *STEER* and COAV101B12302 *STRENGTH*.

Serum urate home monitoring for gout using a Point-of-Care Testing Meter and nurse-led treat-to-target approach: A feasibility study

This research aims to examine the feasibility of home monitoring of serum uric acid for both patients and stakeholders, using a digital home measuring device in conjunction with a nurse-led T2T approach.Primary research question:What is the…

FASTigial: Finding Anatomical SubsTrates of neuropsychological outcome in children with posterior fossa tumors (add-on project on the European CMS study)

The purposes of this study are to uncover- Which clinical and neuroradiological measures can be used to predict the (neuropsychological) outcome and progression of symptoms in children with CMS?We hope that the results will contribute to an overall…

Treating Suicidality Remotely: a randomized controlled trial of brief cognitive behavioral therapy for suicide prevention via chat or phone

Therefore, the main purpose of this study is to investigate whether this online intervention can indeed help prevent suicide attempts. In addition, we want to examine whether the intervention affects thoughts of suicide, quality of life and broader…

Comparing Minimally Invasive Treatments for Pilonidal Disease: LA POPA trial (Laser And Pit-picking OR Pit-picking Alone)

The main objective of this study is to establish the efficacy of *pit picking with laser therapy* versus *pit picking alone* on both short and long-term outcomes.

Ultrasound imaging as a new tool for assessment of respiratory muscle structure and function in patients with Duchenne Muscular Dystrophy

Primary objective: To quantify the degree of respiratory muscle degeneration and dysfunction using ultrasound in patients with DMD over a wide age range; from early childhood to adults.Secondary objective: To determine the association between…

SCreen & INtervene
Neurobiological assessments in screening and individual treatment decisions in the forensic youth setting.

Over the last decades, much fundamental knowledge has been obtained about the neurobiology of antisocial behavior on group level. The main aim of the current study is to translate this group-level knowledge to individualized, developmental profiles…

Optimization of remote assessment in cochlear implant recipients.

A future project will focus on a new platform for at-home monitoring and testing of cochlear implants, developed by one of the manufacturers of cochlear implants, Advanced Bionics (AB). Their Research Application allows for clinicians to read out…

Cohort multiple Randomized Controlled trial in pediatric asthma: infrastructure to assess the long- and short-term effects of (eHealth) interventions

CIRCUS cohort:This study aims to provide a framework to assess the effect of (eHealth) interventions and generate short- and long-term data on clinical and patient-reported outcomes of asthmatic children using a cohort multiple Randomized Controlled…

Growing Up Together in Society (GUTS) - Rotterdam Cohort

The current study describes the Rotterdam Cohort (i.e., a sub-project) of the larger Growing Up Together in Society Project, which has the aim to examine combined social contextual, biological, and behavioral mechanisms that drive the transitions…

Model Informed preciSion doSIng tO iNdividualise and optimize pharmacotherapeutic treatment (MISSION) - Appendix 1: Prospective evaluation of Model informed precision dosing (MIPD) for tacrolimus and mycophenolic acid in paediatric patients with a kidney disease

Overall objective:To improve the existing drug prescribing and treatment monitoring process.Objective appendix 1: To prospectively evaluate the predictive performance of the MIPD strategy for tacrolimus and mycophenolic acid in paediatric patients…

Investigating the benefit and relevance of the automatic classifier Autosense Sky in various complex acoustic conditions in CI children

Gaining insight in the potential benefit and relevance of the automatic classifier in various acoustic conditions for children using Advanced Bionics Sky CI(s).

High intensity interval training in patients with a right ventricle to pulmonary artery conduit

Primary Objective: To assess the efficacy, expressed by change in peak oxygen consumption obtained with cardiopulmonary exercise testing (CPET), of a HIIT program in patients with an RV-PA conduit.Secondary Objective(s): To determine predictors for…

Secondary Manifestations of ARTerial diseases in the Young with a chronic condition

The primary objective of this study is to establish the effect of cardiovascular risk factors on preclinical atherosclerosis in children with a chronic condition, and follow this up over time.

SUicidality and Needs among people Seeking EuThanasia based on psychological suffering (SUNSET)

1. To gain insights into care needs with regard to death wishes, death thoughts and suicidality of people requesting euthanasia due to psychological suffering;2. To gain insights into characteristics of death wishes, death thoughts and suicidality…