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A Multicenter, Multinational, Longitudinal Clinical Assessment Study of Subjects with Mucopolysaccharidosis IVA (Morquio Syndrome).

The objective of the study is to quantify endurance and respiratory function in subjects with MPS IV A and to better characterize the spectrum of symptoms and biochemical abnormalities in MPS IV A disease over time.

Pilot study on effectiveness and safety of Shape Memory Scoliosis Correction Instrument

Primary Objectives:• show effectiveness of the SMSC instrument in 3D correction of the spine prior to placement of the implants.The hypothesis is that to achieve de-rotation of the spine without failure of the pedicle screws, de-rotation has to be…

An Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects with Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) (Extension of Study RET IRD 01)

• To evaluate the safety of up to 3 additional courses of oral QLT091001 administered once daily for 7 days in subjects with LCA or RP caused by RPE65 or LRAT gene mutations who have been treated previously with a single 7-day course of QLT091001 in…

Treatment study for patients with acute promyelocytic leukemia under 21 years of age

The study aims to limit the use of anthracyclines and to reduce the dose of ATRA. Another aim is to stratify treatment by risk group: standard risk - WBC <10 x 109/l : high risk - WBC >=10 x 109/l. Furthermore this study aims to…

An explorative prospective observational case-control pilot of EEG registration of cortical spreading depression in patients aged 4 years or older, with an acute migraine attack admitted at the headache outpatient service of Medisch Spectrum Twente

Primary Objective: To measure cortical spreading depression with full-band EEG during the migraine attack at patients aged 4 years or older. Secondary Objective(s): -To explore (additional) signal analysis techniques for detecting CSDs or infra slow…

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…

ADOLESCE-NT: More rapid deterioration of renal function after renal transplantation in adolescence: effect of the endocrine system on immunological and pharmacokinetic factors

Answering the question if there are any associations between hormonal status in adolescence on the one hand, and immunological reactivity and pharmacodynamics on the other hand; and if such associations can explain the more rapid deterioration of…

Dorsal spondylodesis in adolescent idiopathic scoliosis: proximal fixation with screws versus claw construct

The main objective is to compare the coronal Cobb angle correction of proximal hook claw fixation versus proximal pedicle screw fixation after two years of follow-up. The secondary objectives of the study are: comparison of coronal Cobb angle…

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.

Fatigability in Spinal Muscular Atrophy

To determine the reproducibility and validity of specific outcome measures for skeletal and respiratory fatigability in subjects with SMA.

Bronchiolitis obliterans syndrome and other allo-reactive lung syndromes
after allogeinic hematopoietic stem cell transplantation
Is early prediction of bronchiolitis obliterans possible by using biomarkers possible?

In this study we would like to find new biomarkers in BOS after haematopoietic stem cell transplantation (HSCT), that may lead to earlier identification of BOS than a lung function decline.

A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Cystinosis.

The objective of this pivotal study is to assess the long-term safety, tolerability, pharmacokinetics and pharmacodynamics of RP103 in pediatric and adult patients with nephropathic cystinosis. Results of this Phase 3 study will be used to support…

Assessment of real life and trained inhalation of Tobramycin Inhalation Powder (TIP) and Tobramycin Inhalation Solution (TIS) TIPTIS study.

Primary objective of the study is to record TIP inhalations on video at home to study the association between inhalation manoeuver and cough. Secondary objectives are: To study the inhalation maneuvers of patients using TIP in the home situation; to…

Hypnotherapy for adolescents and adults with inflammatory bowel disease and symptoms compatible with irritable bowel syndrome.

To study the effectiveness of gut-directed hypnotherapy in the treatment of IBS-like symptoms in patients with inflammatory bowel disease.

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…

The effectiveness of Pivotal Response Treatment in children and adolescents with autism spectrum disorder: a randomized clinical trail

Primary Objective: The main objective is to investigate the effectiveness of PRT compared to CAU in school-aged children and adolescents with ASD.The primary research question is:1) Directly after treatment and at follow-up, what is the…

Evaluation of the CAP-value for quantitating liver steatosis in obese children using 1H-MRS as reference standard

To investigate the correlation between the CAP-value measured with the FibroScan® device and liver fat percentage measured with 1H-MRS in a well-characterized cohort of obese children at risk of having fatty liver disease.

Closing the preventive care chain for overweight children: evaluation of the implementation of an integrated, locally aligned approach, enforcing primary care.

Get insight in the implementation and effectiveness of the integrated preventive care chain for overweight children in 's-Hertogenbosch. With the results we can optimize the support for overweight children and their parents. .

Intrathecal baclofen treatment in dystonic cerebral palsy: a randomized clinical trial

Objectives: The primary aim of this study is to provide evidence for the effect of ITB treatment on the level of activities in dystonic CP patients.

Executive and social functioning in early- and continuously-treated patients with PKU: A follow-up study 10 years later

The main objective of this study is to examine the executive, social-cognitive, and social functioning and behaviour of 7-30 year old PKU patients in relation to history of treatment and treatment adherence. It is expected that there will be…