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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.

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…

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…

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…

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.

Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA

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…

Intra-subject variability in pain scoring and the consequences for analgesia treatment

In the current study we want to further explore the predictability of pre-treatment pain variability on the probability to experience pain relief. Knowledge on the understanding of individual differences in analgesic properties of a drug is of…

Effect of whey protein-enriched enteral nutrition in addition to standardized exercise training on the PREServation of MUScle function in critically ill patients: a randomized controlled trial

First we want to determine whether early high protein intake, using an enteral whey protein supplement, in addition to a standardized exercise training program and standard enteral nutrition preserves: a) in vitro skeletal muscle function in…

A Prospective Natural History Study of the Progression of Physical Impairment, Activity Limitation and Quality of Life in Duchenne Muscular Dystrophy (DMD)

•To characterize the natural history and progression of DMD to help inform the design of future studies•To capture biomarkers of safety and disease progression•To provide comparative data for the development of rare exons for which formal controlled…

A randomized, double blind, placebo controlled, multi-centre study to assess the pharmacodynamics, pharmacokinetics, safety and tolerability of BYM338 in chronic obstructive pulmonary disease patients with cachexia (CBYM338X2204)

Primary: Assess the effect of i.v. infusions of BYM338 on muscle volume of the thigh (assessed by MRI) at 4, 8, 16 and 24 weeks, compared to placebo, in COPD patients with pulmonary cachexia.Secondary: Effect on 6-minute walk test, safety and…

Non-invasive assessment of skeletal muscle involvement in Duchenne muscular dystrophy

To develop non-invasive markers for tissue changes in skeletal muscle using MR methods