| Literature DB >> 28884975 |
Sarah J Hart1, Jeannie Visootsak2, Paul Tamburri2, Patrick Phuong2, Nicole Baumer3,4,5, Maria-Clemencia Hernandez6, Brian G Skotko5,7, Cesar Ochoa-Lubinoff8, Xavier Liogier D'Ardhuy6, Priya S Kishnani1, Gail A Spiridigliozzi9.
Abstract
Although an increasing number of clinical trials have been developed for cognition in Down syndrome, there has been limited success to date in identifying effective interventions. This review describes the progression from pre-clinical studies with mouse models to human clinical trials research using pharmacological interventions to improve cognition and adaptive functioning in Down syndrome. We also provide considerations for investigators when conducting human clinical trials and describe strategies for the pharmaceutical industry to advance the field in drug discovery for Down syndrome. Future research focusing on earlier pharmaceutical interventions, development of appropriate outcome measures, and greater collaboration between industry, academia, advocacy, and regulatory groups will be important for addressing limitations from prior studies and developing potential effective interventions for cognition in Down syndrome.Entities:
Keywords: Down syndrome; clinical trials; cognition; interventions
Mesh:
Year: 2017 PMID: 28884975 DOI: 10.1002/ajmg.a.38465
Source DB: PubMed Journal: Am J Med Genet A ISSN: 1552-4825 Impact factor: 2.802