This is a first-in-human (FIH) study of RG6496 that will assess the safety and tolerability of single-ascending doses of RG6496 administered to huntington's disease gene expansion carriers (HDGECs). The study consists of two parts: Part 1 \[single-ascending dose\] followed by Part 2 \[open-label extension (OLE)\].
The goal of this clinical trial is to test if the drug SKY-0515, an oral medication, can lower harmful proteins linked to Huntington's Disease (HD) and improve the symptoms of participants with HD. This study includes men and women aged 25 and older who have HD confirmed by genetic testing …
iMarkHD is an adaptive, longitudinal positron emission tomography (PET) and magnetic resonance (MR) imaging study in Huntington's disease (HD) that aims to assess abnormal molecular, functional, and structural changes in participants' brains, ranging from several years before symptom onset to the advanced symptom stage. The study will be conducted over …
Huntington's disease is a rare and fatal monogenic neurodegenerative disorder whose molecular origin is an expansion of CAG triplets within the first exon of the Huntingtin gene. Although a growing number of emerging therapies are in clinical trials, there are no proven neuroprotective or curative treatments approved by the health …
Lewy body disease (LBD) is a neurodegenerative disorder that causes cognitive and behavioral problems. However, it is difficult to diagnose, and its pathophysiology is still not fully understood. The usual diagnostic criteria for LBD have excellent specificity but poor sensitivity. In particular, we lack paraclinical tests that can make a …
Neurodegenerative diseases are a major health concern due to their growing societal implications and economic costs. The identification of early markers of pathogenic mechanisms is one of the current main challenges. The gut-brain axis has become a primary target because of its transversal role across the neurodegenerative spectrum and its …
MEND-HD is a longitudinal study evaluating the feasibility of passive monitoring of gait and chorea in patients with HD and the meaningfulness of these outcomes for patients with HD and their care partners/support persons. Participants will take part in four virtual visits with study investigators to answer survey questions on …
Lewy body diseases (LBDs) represents a group of neurodegenerative disorders characterized by the abnormal accumulation of aggregates of α-synuclein protein leading to the formation of Lewy bodies (LB) and Lewy neurites resulting in cell death. LBDs consists of two major clinical entities - Parkinson's disease (PD) and dementia with LB …
The goal of this clinical trial is to learn whether a personalized, multi-component, virtual speech language treatment program can improve communication and quality of life for adults with primary progressive aphasia (PPA) and their primary communication partners. The study will enroll participants who speak English and/or Spanish. The main questions …
AD afflicts over 5.5. million Americans and is one of the most expensive diseases worldwide. In AD the variant in which language functions are most affected are referred to as 'logopenic variant Primary Progressive Aphasia' (lvPPA). Language deficits dramatically impair communication and quality of life for both patients and caregivers. …
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