90 results
The objectives of this study are to:• Evaluate the safety, tolerability, and immunogenicity of long-term treatment with intravenous (IV) ATYR1940 in adult patients with facioscapulohumeral muscular dystrophy (FSHD) previously enrolled in clinical…
To evaluate the long-term safety, tolerability, and efficacy of ABT-494 in RA subjects who have completed Study M13-550 or Study M13-537 Phase 2 RCT with ABT-494.
Primary objective:Evaluate the safety, tolerability, pharmacokinetics (PK), and immunogenicity of multiple doses of intravenous (IV) ATYR1940 in adults 18 to 65 years of age, inclusive, with FSHDSecondary objective:Explore pharmacodynamic (PD)…
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 purpose of this study is to find out which common problems occur in the HME patient population. Furthermore the study will investigate which factors are associated with the observed problems. This is barely investigated in the current literature…
The aim of our study is to further elucidate the role of genetic factors in the pathogenesis of CTS by genetic and functional research.-Objective 1: Unravel the mutational spectrum of genes in patients with idiopathic CTS-Objective 2: Evaluation of…
By making this design study we wish to contribute to profound research on percutaneous gastrocnemius lengthening for for children with an equinus contracture and prevent publication bias for this open-labelled randomized trial.
The objective of this study is to determine the optimal myo-electric prosthesis training for obtaining the largest transfer effect. Here fore we plan to execute three experiments. At first it needs to be revealed which trainingtasks leads to the…
The objective of this study is to determine the surplus value of mental imagery on myo-electric prosthesis training Here fore we plan to execute two experiments. The goal of the first experiment is to test if intermanual transfer effects of able-…
In one FOA family we have detected a deleterious mutation in a compelling gene which co-segregates with the FOA phenotype in the family. This mutation is, therefore, is likely to be causal to the early onset OA. The objective of the study is:a) To…
The objectives of this study are:1) to quantify the muscle symptoms, muscle complaints, limitations in daily living, fatigue, mood disorders, and quality of life in patients with Brody's syndrome.2) determining the genetic background of Brody…
The primary objective is to compare the safety and efficacy of multiple doses of ABT-494 versus placebo in moderately to severely active RA subjects on stable background MTX therapy with inadequate response or intolerance to anti-TNF biologic…
The primary objective is to identify (epi)genetic and environmental disease modifying factors that contribute to the variable clinical phenotype of FSHD, in order to support the future development of new treatment strategies for FSHD. The secondary…
1. The effect of Losartan on the joint excursions and the Hardening of the skin in a patient with SSS.2. Gain insight in the pathogenesis of SSS3. Gain insight in the functional effect of Losartan in SSS.
The objective of this study is to establish the effects of transfer of learning in children with upper limb deficiencies after practising with an upper limb prosthetic simulator.In children, transfer of learning effects have hardly been studied. The…
2.1 Primary Objectives: 1) To test the feasibility of the six minute assisted leg and arm cycling test in patients aged 6-18 years with Duchenne muscular dystrophy (DMD), Beckers muscular dystrophy (BMD), Limb Girdle muscular dystrophy (LGMD),…
the aim of the current research project is to investigate the effect of the mirror box illusion on movement performance, neuromuscular activation and proprioception in children with SHCP. The following objectives are: to determine the effect of the…
To investigate whether children with OI who use a wheelchair (for long distances) can reliably perform a valid shuttle ride test in order to assess the levels of health-related fitness in this group.
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.
Primary Objective: To determine the prevalence of endo- and extranasal deformities, problems with nasal functioning and satisfaction with nasal appearance in patients with TCS.Secondary Objective: To adjust and improve the treatment of patients with…