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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…

A randomized controlled trial on the effects of periconceptional and prenatal folic acid supplementation on congenital anomalies and preterm birth

In the proposed study, two main issues will be addressed: first, it will be studied whether a high dose (4.0 mg) of FA supplementation periconceptional has an added value over the now recommended low dose of 0.4 mg in the prevention of congenital…

Targeting Antibiotics to Pseudomonas Aeruginosa in Small airways (TAPAS) study in patients with cystic fibrosis

Primary objective:To compare the change in small airways obstruction (FEF75%) in patients with CF when inhaling one ampule of inhaled tobramycin with the Akita® compared to standard of treatment (twice daily nebulization of one ampule using standard…

Desmopressin and FVIII concentrate combination treatment in non-severe hemophilia A patients undergoing minor interventions

To show a reduction in FVIII-concentrate consumption with perioperative desmopressin and FVIII concentrate combination treatment compared to FVIII concentrate monotherapy, without decreasing the effectivity of treatment.

Treatment of Osteogenesis Imperfecta with Parathyroid hormone and Zoledronic acid

This study has been transitioned to CTIS with ID 2024-519705-36-00 check the CTIS register for the current data. The primary objective will be to investigate if a two-year course of TPTD followed by antiresorptive treatment with a single infusion of…