Journal
ALZHEIMERS RESEARCH & THERAPY
Volume 6, Issue 5, Pages -Publisher
BIOMED CENTRAL LTD
DOI: 10.1186/s13195-014-0061-6
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Funding
- NICHD NIH HHS [R01 HD064993] Funding Source: Medline
- EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH & HUMAN DEVELOPMENT [R01HD064993] Funding Source: NIH RePORTER
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Background: Adults with Down syndrome develop Alzheimer's disease neuropathology in an age-dependent manner. This unique feature provides an opportunity to test interventions targeted for prevention of Alzheimer's disease neuropathology and dementia in Down syndrome. Discussion: In considering clinical trial designs, however, there are several challenges that we believe will be critical to examine further. These include: accuracy in dementia, mild cognitive impairment and preclinical Alzheimer's disease diagnoses in Down syndrome; clinical trial outcome measures appropriate for individuals with Down syndrome; in vivo imaging outcome measures (and practical considerations); and contributions of medical co-morbidities to disease progression. Also, when studies are designed, the molecular target may appear to be obvious (for example, targeting beta-amyloid pathology), but chromosome 21 has over 200 additional genes that could influence both positive and negative clinical trial outcomes. Summary: Observational longitudinal studies of aging in Down syndrome will be critically important as there is a need to establish sensitive clinical outcome measures and understand the consequences of gene overexpression in relation to specific interventions.
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