209 results
Primary:The primary objectives of the study are to estimate and rank-order the longitudinal standardized mean changes over 6 months and over 12 months, for a set of novel outcome measures administered to subjects with ALS, in order to identify…
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…
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…
Primary:The purpose of this study is to evaluate the safety, tolerability, orally administered LMI070 in patients with Type 1 SMA.pharmacokinetics (PK), pharmacodynamics (PD) and efficacy; and to estimate the Maximum Tolerated Dose (MTD) and optimal…
To evaluate whether neuromuscular ultrasound discriminates between patients with and without ICU-AW.To detect differences between patients that do and do not have ICU-AW in order to determine relevant cut-offs which can be used in future diagnostic…
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…
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…
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…
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…
The primary objectives of this study are (a) to evaluate reliability and precision of an instrumented spasticity assessment device (ISA) in children with cerebral palsy and adults with spastic paresis (MS and stroke patients) and (b) to validate ISA…
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…
Main objective: - Obtain long-term clinical follow-up data in PLS patients to investigate the reliability of the diagnosis PLS. - Obtain data on cognitive functioning to investigate the possible relation between PLS and FTD.Secondary objectives: -…
To evaluate measurement properties and feasibility of the self-report Northwestern Ego-Integrity Scale in patients with ALS (or its variants PMA and PLS)
To determine the reproducibility and validity of specific outcome measures for skeletal and respiratory fatigability in subjects with SMA.
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…
a. To establish the effects of (1) body weight support (2) treadmill speed, and (3) level of movement guidance provided by the exoskeleton on the neuromuscular control of the lower extremities.b. To establish the interactions between the above…
The present protocol addresses these issues, with a systematic approach where we first assess optimal settings for BCI, and then test the quality of BCI application in adequately sized groups of subjects. The whole project is aimed at obtaining a…
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…
- To study the clinical characteristics and disease course of patients with LEMS with or without SCLC- To identify the characteristics of the humoral or cellular immune response that are associated with the prolonged survival of patients with SCLC…
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…