87 results
The primary objective of TRIHEP 3 is to evaluate the efficacy of triheptanoin in (i) increasing the short term energy response in the metabolic profile of the brain of early affected HD patients, as captured by 31P-MRS, and (ii) slowing atrophy in…
Primary Objective: The primary objective of this study is to evaluate safety and tolerability of pridopidine in patients with HD.Secondary Objectives: The secondary objectives of the study are to assess the effects of long-term, open-label dosing…
This study aims to demonstrate the clinical efficacy of patisiran and to establish the safety of chronic dosing in ATTR patients with FAP.The primary objective of the study is to determine the efficacy of patisiran by evaluating the difference…
Primary Study Objective: The primary objective of this study is to assess the efficacy of laquinimod 0.5 mg and 1.0 mg qd in patients with HD after 12 months of treatment using the UHDRS-TMS.Secondary Study Objectives:• To assess the effect of…
The main purpose of this study is to find out whether EMDR is a safe and effective treatment to reduce the symptoms of PTSD in people with mild intellectual disability and borderline intellectual functioning. In addition, it is expected to reduce…
The objective of the study is to investigate if it is possible to predict the clinical outcome of a patient with SCN1A related epilepsy based on the findings of advanced genotyping. We aim to answer the following questions:1. Can clinical outcome in…
We aim to conduct a five year follow-up amongst the X-ALD carriers and assess their current symptomatic and biochemical status. This data will provide new insights in the progression of the disease in carriers. Moreover we would like to validate a…
To determine the feasibility of rs-fMRI in neonates undergoing a planned diagnostic MRI session following perinatal asphyxia. To assess intra-session reproducibility (test-retest reliability) of the rs-fMRI technique in neonates with asphyxia.
Main objectives:To determine differences in NeuroCart profiles in different sub-groups based on cognitive testing and CSF biomarker profiling (i.e. cognitively normal, pre-symptomatic AD, prodromal AD, MCI, mild AD).To characterize a cohort of…
Primary objective: The main objective of this pilot study is to gain more insight into the effects of tVNS: to assess if the ABVN and consecutively the NTS are really stimulated by tVNS. Therefore this study investigates the effects of tVNS on the…
Obtain information regarding the movement abilities and muscle strength in persons with several neuromuscular diseases, which can be used to develop a motion controlles arm suport for daily use and training of arm function.
The objective is to assess the gait characteristics of adults with intellectual disabilities by other causes than Down syndrome, and without diagnosed neurological conditions (Parkinson's disease, cerebrovascular accident, dementia, and…
2.1 Primary Objectives• Evaluate the safety and tolerability of 2 fixed doses of FRM-0334 (300 and 500 mg daily in 2 sequential periods) over 28 days in subjects with prodromal to moderate FTD-GRN• Assess the PD effects of FRM-0334 on the change…
To gain insight (1) into psychological functioning of couples where one partner is carrier of HD and who have PGD child(ren); and (2) into the family interactions in these families.
The main objective of this prospective cohort study of patients with proximal SMA in the Netherlands is to investigate the function of the neuromuscular junction by means of repetitive nerve stimulation (RNS).
To determine the safety and efficacy of Dimebon in patients with mild-to-moderate Huntington Disease
(1) Identification of novel loci and genes in which mutations are responsible for the development of inherited epileptic syndromes. (2) Establishing genotype-phenotype correlations; defining the yield of mutation screenings in specific patient…
1) What is the long-term course of tics and comorbid OCD, ADHD and Quality of life in patients with GTS? 2) What are determinants of favourable versus unfavourable course?
To evaluate associations between specific characteristics of DA GMs around 3 months CA and daily function at 8 years.
To evaluate the effect of additional foot support during sitting and the effect of forward tilting of the seat surface on 1) postural adjustments during reaching and 2) the quality of reaching movement in school age children with CP.