Developing Neurophysiological Outcome Measures for Treatment Assessment in Niemann-Pick type C (NPC) Disease
Project Description:
Developing brain measures that can be used to track disease progression and treatment efficacy in Niemann-Pick Type C by looking at cognitive neuroscience measures aimed at testing basic sensory processing and multisensory integration. To do this we use behavioral tasks and scalp-recorded electroencephalography (EEG) to measure the brain's automatic response to sensory stimuli. Basic sensory processing is analyzed in the form of evoked potentials, which represent the brain's activity that is time locked to the sensory stimulus and filtered for noise/random activity. Multisensory integration represents the brain's ability to combine sensory information from different modalities to produce something that is above-and-beyond what either modality can do independently.
The measures being examined in this project may provide important insight and have strong clinical implications as we are directly assaying brain function. This can provide an objective marker against which neurocognitive function can be compared. Most significantly though, changes in these measures would necessarily come "online" before changes in behavior or other clinical measures, as any new behavior (be it adaptive or detrimental) must be preceded by a change in the brain (e.g. learning or deterioration). As such, this experiment holds promise in aiding to develop markers for disease progression and against which to test treatment efficacy or toxicity, as they are closer to neural mechanisms which are being altered by disease and treatment agents.
Core Function(s):
Performing Research or Evaluation
Area of Emphasis
Health-Related Activities
Target Audience:
Children/Adolescents with Disabilities/SHCN
Unserved or Under-served Populations:
Specific Groups
Primary Target Audience Geographic Descriptor:
National
COVID-19 Related Data:
N/A
