68 results
The aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with the study drug. The information gained from this study is expected to further characterize the efficacy and safety of BMN 044 over a…
The purpose of this study is to assess the safety, tolerability and efficacy of long term drisapersen in subjects with DMD.
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…
To evaluate the effects of intravenous iron suppletion in iron deficient PAH patients.Amendment: to compare exercise capacity and isolated muscle strength in IPAH patients and healthy controls
The objective of this feasibility study is compare the smart AFO + standardized shoes to subjects* own AFO + own shoes. Subjects* experiences with the orthotic devices will be evaluated and points for improvement will be asked. Subjects* performance…
Looking into the effects of raw vs boiled eggs vs isocaloric low-protein meal on muscle growth.
The primary objective of this study is to compare respiratory muscle strength values obtained using volitional techniques with values obtained using non-volitional techniques. The secondary objective is to compare the change for these two sets of…
Primary objective: To assess the efficacy of BMN 053 at recommended dosing regimen after 48 weeks treatment in ambulant subjects with Duchenne muscular dystrophy.Secondary objectives: - To assess the safety and tolerability of BMN 053 after single…
In this project we use skeletal muscle biopsy tissue from DM1 patients and controls to isolate pericytes. These muscle-derived mesodermal cells can be genetically corrected by the novel powerful CRISPR-Cas9 technique, which will remove the disease…
The aim of this study is to conduct a pilot study of weight-supported ambulation training in ICU patients and to assess its feasibility of the use in daily practice, and to obtain information for the development of a randomized clinical trial for…
Main objective: The main objective of this open-label, single arm study is to further characterize the safety, tolerability and effectiveness profile of olesoxime in SMAThe primary safety objective for this study is as follows:To evaluate the safety…
To develop non-invasive markers for tissue changes in skeletal muscle using MR methods
In recent studies physical training against resistance appeared to be the most successfull to prevent sarcopenia. Because of the bad physical condition of the elderly during a hospital admission resistancetraining can't be applied. A Pilot…
The objective of this prospective study is to answer the question: "Leads isolated dynamic resistance training of the lumbal extensor muscles to decreased multifidus atrophy and will MRI show an increasing multifidus cross sectional area and…
The objective of this pilot study is to demonstrate that NAVA ventilation is superior compared to pressure support and pressure control ventilation in work of breathing (expressed as pressure-time product) patient ventilator synchrony and other…
To determine whether 3D arm training with arm-support is effective in delaying the loss of arm functions in daily activities from an early age onwards in boys with DMD.
The objective of the study is to investigate the effectiveness of a combined intervention on the disabilities, self-efficacy and long-term employability of workers with complaints of the arm, neck and / or shoulder. Through a self-management program…
Primary Objective: Is there reduction in pain using an abduction brace in patients undergoing arthroscopic cuff repair? Secondary Objectives: - Are there differences in function and recovery of the cuff, determined using questionnaires and echo?- Is…
T\o evaluate the pharmacokinetics, safety and tolerability of the research medication
To investigate the relationship between self-reported disability, performance based disability and psychological factors in patients with complaints of whiplash.