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Prevention of renal and bladder damage in children with spina bifida by means of early injections with Botulinum-Toxin-A (Botox): a pilot study.

- Can early Botox-injections in the bladder of patients with meningomyelocèle (MMC) prevent deterioration of the bladder and renal function?- Can early Botox-injections in the bladder of patients with meningomyelocèle (MMC) decrease the need for an…

Ten years of statin therapy started in childhood: efficacy, safety, compliance and tolerability in patients with familial hypercholesterolemia (AfterTen-study)

This study will evaluate the effect of approximately 10 years of pravastatin treatment on IMT in young adults with FH. Furthermore, safety parameters and psychological aspects with respect to early treatment initiation will be investigated.

Radicality of (simple) mastectomy: residual breast tissue

Riskreduction of developing breast carcinoma by means of better surgical techniques (removing all normal breast tissue).

A randomised controlled trial on the effects of an individual tailored lifestyle intervention to minimise the cardiovascular disease risk of individuals with Familial Hypercholesterolemia (FH)

The study aims at examining the effect of an individual tailored lifestyle intervention to reduce the cardiovascular disease risk in people with FH.Secundary, the study aims at promoting a healthy lifestyle (no smoking, proper nutritional pattern,…

Improving risk assessment in consanguineous couples by means of SNP analysis

The objective of this research is to develop a method which can identify among all consanguineous couples the ones which are at high risk.

Fifteen years of statin therapy started in childhood: efficacy, safety, compliance and tolerability in patients with familial hypercholesterolemia

The aim of this study is to evaluate the efficacy, safety, compliance and tolerability of 15 years of pravastatin therapy initiated during childhood in young adult FH patients.

An exploratory, open label, multiple dose, multicentre phase I/II trial evaluating safety and efficacy of prenatal and/or postnatal infusion of allogenic expanded fetal mesenchymal stem cells for the treatment of severe Osteogenesis Imperfecta compared with historical and untreated prospective controls.;In the Netherlands patients will not be treated. Retrospective and prospective control patients will be included

This study has been transitioned to CTIS with ID 2023-504593-38-00 check the CTIS register for the current data. The aim of this trial is to infuse multiple doses of human 1st trimester liver-derived MSC for the treatment of severe OI to determine…

Early TUbectomy with delayed oophorectomy to improve quality of life as alternative for risk reducing salpingo-oophorectomy in BRCA1/2 gene mutation carriers

To evaluate RRS after childbearing with delayed RRO as an alternative for RRSO in BRCA1/2 gene germline mutation carriers. We hypothesize that delay of menopause leads to an improvement of quality of life and sexual functioning, and a decrease in…

Electromechanical profiling of arrhythmogenic substrates and triggers in the long-QT syndrome

To identify arrhythmogenic electromechanical risk profiles in LQTS patients; in baseline conditions and during provocation; the recognition of risk profiles will improve risk stratification for sudden cardiac death.

TUbectomy with delayed oophorectomy as alternative for risk-reducing salpingo-oophorectomy in BrcA-Women to assess the Safety of Prevention: the TUBA-WISP II study

To evaluate RRS with delayed RRO as an alternative for RRSO in BRCA1/2 gene germline mutation carriers. We hypothesize that RRS with delayed RRO leads to an equal ovarian cancer incidence when compared to RRSO.