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A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy

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…

An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy.

The purpose of this study is to assess the safety, tolerability and efficacy of long term drisapersen in subjects with DMD.

Muscle relaxation properties in myopathies with positive muscle phenomena: a study using transcranial magnetic stimulation

To quantify muscle relaxation properties of fingerflexors using transcranial magnetic stimulation in patients with different myopathies to develop a screening diagnostic tool to assess who does (not) suffer from an underlying myopathy.

A phase IIb, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple subcutaneous doses of BMN 045 (previously known as PRO045) in subjects with Duchenne muscular dystrophy

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…

Optimizing the amount of dietary protein to maximally stimulate post-exercise muscle protein synthesis in elderly men.

To define the amount of dietary protein required to optimally stimulate post-exercise muscle protein synthesis in the older population.To assess whether co-ingesting 1.5 g of free leucine (contained within 15 g dairy protein) along with 15 g protein…

Ammendment of protocol: Intravenous iron treatment in iron deficient patients with idiopathic pulmonary arterial hypertension. Title amendment: Skeletal muscle strength in healthy persons and patients with pulmonary hypertension

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

A randomized, double-blind, placebo-controlled, multicenter, parallel group, dose-finding, pivotal, phase IIb/III study to evaluate the efficacy, safety and tolerability of intravenous BYM338 at 52 weeks on physical function, muscle strength, and mobility and additional long-term safety up to 2 years in patients with sporadic inclusion body myositis (CBYM338B2203)

Primary: to demonstrate that at least one dose regimen of BYM338 in sporadic inclusion body myositis patients will increase the distance traveled as measured by change from baseline at Week 52 of the 6 minute walking distance test relative to…

Feasibility study of a smart ankle foot orthosis in people with paretic ankle muscles

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…

The impact of short-term bed-rest on skeletal muscle mass and insulin sensitivity in healthy, young men

To investigate the impact of 7 days of bed-rest on skeletal muscle mass, strength and whole-body insulin sensitivity in healthy, young men.

Diaphragm-protective mechanical ventilation in critically ill patients: A proof of concept study.

Primary objective of this study is to assess the feasibility of using a monitoring and a titration algorithm to prevent inactivity and excessive activity of the diaphragm. This will be determined by comparing the fraction of breaths where diaphragm…

The effect of resveratrol on metabolism and cardiovascular risk profile in patients with chronic obstructive pulmonary disease (COPD)

The overall objective is to investigate the efficacy of resveratrol in modulating metabolism and CVD risk profile in patients with COPD. The primary objective is to investigate the effect of resveratrol on CRP as clinical marker of low grade…

Evaluation of boiled vs raw eggs for stimulating post-exercise muscle protein synthesis

Looking into the effects of raw vs boiled eggs vs isocaloric low-protein meal on muscle growth.

Coordination of trunk, head and arm movements during functional tasks, in healthy children and children with Duchenne muscular dystrophy, spinal muscular atrophy and cerebral palsy.

First objective: gain insight in the coordination of trunk, head and arm during functional tasks in healthy children and compare this with the coordination in children with DMD, SMA and CP. Second objective: investigate if newly developed supportive…

A Study of Respiratory Muscle Strength, including Effort-Independent Measures, in Subjects with Late-Onset Pompe Disease

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…

A Phase I/II, open-label, dose escalating with 48-week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously knoen as PRO053) in subjects with Duchenne muscular dystrophy

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…

Development of an Autologous Myogenic Cell Therapy against the Neuromuscular Phenotype of Myotonic Dystrophy type 1.

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…

Effect of Fecal Transplantation on Satiety, Sarcopenia, Inflammation and Chemotherapy Toxicity in patients with Metastasized Oesophageal and Gastric Cancer.

Primary objective:Effect of fecal transplantation (from healthy obese donors) on faecel microbiota composition in relation to satiety (questionnaires, biomarkers) and metabolism (REE ) in patients with metastasized or locally advanced oesophageal or…

Steps to Recovery; Body weight-supported treadmill training for patients in the intensive care unit: a feasibility study

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…

ROBot-aided system Identification: a novel tool for diagnosis and assessment in Neurological rehabilitation: *ROBIN*

Primary objective of this study is to assess the reliability, validity, and diagnostic value of the neuromechanical parameters of joint impedance estimated by nonlinear SIPE technology in patients with a spastic paresis.

Acceptability and feasibility of exercise therapy in an aquatic environment for patients after burn injury

To investigate the acceptability and feasibility of exercise therapy in an aquatic environment by patients after burn injury.