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Spinal Muscular Atrophy, SMN protein and Genetics; A population based study on spinal muscular atrophy in the Netherlands to explore the prevalence of SMA type 1-4 and their severity in the Netherlands by registrating all patients in a so called SMA-database, to elucidate the genetic factors determining disease severity and to determine the value of SMN protein concentrations as a surrogate marker for clinical trials.

The main objective of this prospective population-based study of patients with proximal SMA in the Netherlands is to register SMA patients in the Netherlands in a socalled SMA-database to study prevalence and severity of SMA types 1-4, to perform…

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy of Natalizumab on Reducing Disability Progression in Subjects With Secondary Progressive Multiple Sclerosis, With optional Open-label Extension

Part 1:The primary objective of the study is to investigate whether treatment with natalizumab slows the accumulation of disability not related to relapses in subjects with SPMS. The secondary objectives of this study are to determine in this study…

A placebo-controlled double blind randomized trial to investigate the efficacy and safety of the combination of Penicillin G / Hydrocortisone treatment in ALS patients (PHALS).

The objective is to compare the efficacy and safety of intravenous high-dose penicillin G and hydrocortisone versus placebo in combination with riluzole in the treatment of patients suffering from Amyotrophic Lateral Sclerosis (ALS).

A Phase 3, Multi-National, Double-Blind, Randomized, Placebo-Controlled, Stratified, Parallel Group, Study to Evaluate the Safety, Tolerability and Efficacy of Tirasemtiv in Patients with Amyotrophic Lateral Sclerosis (ALS)

Protocol version Amendment 1, 31-Jul-2015, paragraph 2.1, page 28:Primary objective:The primary objective is to assess the effect of tirasemtiv versus placebo on respiratory function in patients with ALS.Secondary objectives:* Evaluation of…

Influenza vaccination in patients with Myasthenia Gravis

The main objective of this study is to investigate the effectiveness of the humeral immune response after influenza revaccination in patients with MG with acetylcholine antibodies (AChR MG). The secondary objectives are to determine if vaccination…

HOMERUN
Initiation of HOme MEchanical ventilation at home in a selective group of patients with chronic hypercapnic Respiratory failUre in the Netherlands

Patients with chronic respiratory failure currently start their ventilatory support in hospital as stated in national guideline. However, as these patients are severely disabled, a stay at the hospital for several days is experienced by many as very…

The frontotemporal syndrome in amyotrophic lateral sclerosis: screen, impact, imaging and pathology.

The main objectives are: 1. to develop a diagnostic tool for the assessment of the frontotemporal syndrome. 2. To investigate the structural and functional brain networks of ALS patients with and without a frontotemporal syndrome, in order to gain…

A Randomized, Double blind, Placebo Controlled Phase II Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX 113 in Patients with Myasthenia Gravis who have Generalized Muscle Weakness

Primary Objective:* To evaluate the safety and tolerability of ARGX-113.Secondary Objectives:* To evaluate the clinical effect of ARGX-113 using:-Myasthenia Gravis-Activities of Daily Living (MG-ADL) score.-Quantitative-Myasthenia Gravis score (QMG…

In vivo imaging of brain metabolism in ALS

To study the role of brain metabolites and macromolecules in relationship to pathogenesis, structural brain changes and clinical phenotype of ALS. This will reveal underlying molecular mechanisms of pathogenesis, structural brain changes and…

Fatigability in Spinal Muscular Atrophy

To determine the reproducibility and validity of specific outcome measures for skeletal and respiratory fatigability in subjects with SMA.

MR Imaging in Spinal Muscular Atrophy; A pilot study to uncover neuronal network changes

To investigate motor connectivity in vivo in patients with SMA using MR- techniques for brain imaging.We will investigate connectivity of upper motor neurons in patients with SMA compared to disease controls (patients with myopathic disorders) and…

Prevention of depression and poor physical function in older persons with vitamin D supplementation

Primary objectives: 1. Does vitamin D supplementation improve depressive symptoms in older persons? 2. Does vitamin D supplementation improve physical performance and functional limitations in older persons?Secondary objectives: 3. Does vitamin D…

Effectiveness of a standardized physical therapy program in cervical dystonia; a single blind randomized controlled trial.

Primary objectiveThe primary objective is to evaluate the effectiveness of a standardized PT program according to the treatment guideline on improvement of the ability to perform daily life tasks compared to regular PT as add-on treatment to BTX-…

The role of genetic factors in the aetiology of achalasia

Primary:- To identify causal genetic variants and proteomes involved in achalasia and to study their relevanceSecundary:- To build-up a large scale harmonized biobank of achalasia patients, based on combining existing and new data from the European…

*The reproducibility of the energy cost of walking test and the 6-minute walk test to assess the physical strain of walking in post-poliomyelitis syndrome patients'

1. To determine the reproducibility of the ECWT and the 6MWT to assess the physical strain of walking in patients with PPS and in healthy control subjects.2. To evaluate the one-year course of the physical strain of walking among patients with PPS…

Neuromuscular functions in patients with the chronic fatigue syndrome versus patients with Fibromyalgia Syndrome and healthy people.

To investigate whether the CVS patients show the muscle membrane disturbances, as the FMS patients do. That is, are there muscle conduction velocities disturbances in the CVS patients?

Energetic, a self-managment group program aimed at reconditioning and participation in persons with an muscle disease; a randomized controle trial.

To evaluate the effectiveness and cost-effectiveness of Energetic. Secondary objectives include evaluation of barriers and facilitators for implementation of Energetic in other settings.

A novel neurophysiological measurement (MUNIX) in neuromuscular disorders; test-retest reliability and longitudinal follow up measurements in ALS patients

PrimaryTo investigate the inter-rater and intra-rater variability of a novel neurophysiological measurement (Motor Unit Number Index, MUNIX) for estimation of functioning motor units, in healthy controls and ALS patientsSecondaryTo evaluate the…

Effects of levosimendan on diaphragm contractility in healthy subjects

The primary objective of this proof of principle study is to determine the effect of levosimendan on the strength and endurance of the diaphragm in healthy subjects. The secondary objective is to gain insight in the neural activation and efficiency…

Muscle relaxation time as outcome measure for myotonia in patients with non-dystrophic myotonic syndromes (NDMs)

In this study we want to investigate if relaxation time (RT) measured by handgrip myometry and muscle- elastography is a reliable outcome measurement for the quantification of myotonia in NDM patients. Mean RT values en standard deviations of NDM…