9 results
The primary objective is to assess neuropsychological function in X-ALD patients without active cerebral demyelinating disease. Data of minors will be compared to matched healthy controls. Data of adults will be compared to reference values. The…
Primary Objective:To evaluate the effect and sustainability over time of behavioural treatment for sleep problems in children with AS on the actual hours of sleep and sleep behaviour of the children.Secondary Objectives:To evaluate the effect and…
The primary objective is to measure the longitudinal changes of PDE10A enzyme availability in the caudate, putamen, and globus pallidus of Huntington*s Disease Gene Expansion Carrier (HDGECs) by comparison of the follow-up and initial Positron…
Primary objective: - 50% seizure reduction or more at 3 months of dietary treatment .Secondary objectives: -level of ketosis on KD and MAD.-number of patients maintaining >50% seizure reduction on MAD.-number of withdrawals (not able to…
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…
To assess the safety and efficacy of long-term dosing with patisiran in transthyretin-mediated amyloidosis (ATTR) patients with familial amyloidotic polyneuropathy (FAP)
Primary objectives:* To improve the understanding of alterations in the visual pathway in patients with HD in different disease stages.* To investigate possible changes in brain function and structure in rest and in response to visual stimulation in…
* To investigate oculomotor and vestibular activity in individuals with RTT. * To compare oculomotor and vestibular activity with more functional eye gaze and communication skills. * To compare oculomotor and vestibular activity with severity of…
(I) Compare shoulder loads estimated with musculoskeletal simulations, stability and associated muscular effort of subjects with FSHD and healthy controls during the performance of standardized upper extremity tasks with and without an arm support…