Literature DB >> 28884975

Pharmacological interventions to improve cognition and adaptive functioning in Down syndrome: Strides to date.

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.
© 2017 Wiley Periodicals, Inc.

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


  15 in total

1.  Challenges and Opportunities for Translation of Therapies to Improve Cognition in Down Syndrome.

Authors:  Sarah E Lee; Monica Duran-Martinez; Sabina Khantsis; Diana W Bianchi; Faycal Guedj
Journal:  Trends Mol Med       Date:  2019-11-07       Impact factor: 11.951

2.  Short-term memory outcome measures: Psychometric evaluation and performance in youth with Down syndrome.

Authors:  Emily K Schworer; Kellie Voth; Emily K Hoffman; Anna J Esbensen
Journal:  Res Dev Disabil       Date:  2021-12-15

3.  Evaluating Verbal Fluency Outcome Measures in Children With Down Syndrome.

Authors:  Catelyn N Smeyne; Anna J Esbensen; Emily K Schworer; Shequanna Belizaire; Emily K Hoffman; Dean W Beebe; Susan Wiley
Journal:  Am J Intellect Dev Disabil       Date:  2022-07-01

Review 4.  Down syndrome.

Authors:  Stylianos E Antonarakis; Brian G Skotko; Michael S Rafii; Andre Strydom; Sarah E Pape; Diana W Bianchi; Stephanie L Sherman; Roger H Reeves
Journal:  Nat Rev Dis Primers       Date:  2020-02-06       Impact factor: 52.329

5.  A non-mosaic transchromosomic mouse model of down syndrome carrying the long arm of human chromosome 21.

Authors:  Yasuhiro Kazuki; Feng J Gao; Yicong Li; Anna J Moyer; Benjamin Devenney; Kei Hiramatsu; Sachiko Miyagawa-Tomita; Satoshi Abe; Kanako Kazuki; Naoyo Kajitani; Narumi Uno; Shoko Takehara; Masato Takiguchi; Miho Yamakawa; Atsushi Hasegawa; Ritsuko Shimizu; Satoko Matsukura; Naohiro Noda; Narumi Ogonuki; Kimiko Inoue; Shogo Matoba; Atsuo Ogura; Liliana D Florea; Alena Savonenko; Meifang Xiao; Dan Wu; Denise As Batista; Junhua Yang; Zhaozhu Qiu; Nandini Singh; Joan T Richtsmeier; Takashi Takeuchi; Mitsuo Oshimura; Roger H Reeves
Journal:  Elife       Date:  2020-06-29       Impact factor: 8.140

6.  Expressive language sampling as a source of outcome measures for treatment studies in fragile X syndrome: feasibility, practice effects, test-retest reliability, and construct validity.

Authors:  Leonard Abbeduto; Elizabeth Berry-Kravis; Audra Sterling; Stephanie Sherman; Jamie O Edgin; Andrea McDuffie; Anne Hoffmann; Debra Hamilton; Michael Nelson; Jeannie Aschkenasy; Angela John Thurman
Journal:  J Neurodev Disord       Date:  2020-03-24       Impact factor: 4.025

7.  Spoken language outcome measures for treatment studies in Down syndrome: feasibility, practice effects, test-retest reliability, and construct validity of variables generated from expressive language sampling.

Authors:  Angela John Thurman; Jamie O Edgin; Stephanie L Sherman; Audra Sterling; Andrea McDuffie; Elizabeth Berry-Kravis; Debra Hamilton; Leonard Abbeduto
Journal:  J Neurodev Disord       Date:  2021-04-08       Impact factor: 4.025

8.  Context Fear Conditioning in Down Syndrome Mouse Models: Effects of Trisomic Gene Content, Age, Sex and Genetic Background.

Authors:  Md Mahiuddin Ahmed; Aaron Block; Nicolas Busquet; Katheleen J Gardiner
Journal:  Genes (Basel)       Date:  2021-09-28       Impact factor: 4.096

9.  Semantic Verbal Fluency in Youth with Down Syndrome: Analysis of Conventional and Contextual Cluster Formation.

Authors:  Emily K Schworer; Shequanna Belizaire; Emily K Hoffman; Anna J Esbensen
Journal:  Brain Sci       Date:  2021-12-23

10.  Opportunities, barriers, and recommendations in down syndrome research.

Authors:  James A Hendrix; Angelika Amon; Leonard Abbeduto; Stamatis Agiovlasitis; Tarek Alsaied; Heather A Anderson; Lisa J Bain; Nicole Baumer; Anita Bhattacharyya; Dusan Bogunovic; Kelly N Botteron; George Capone; Priya Chandan; Isabelle Chase; Brian Chicoine; Cécile Cieuta-Walti; Lara R DeRuisseau; Sophie Durand; Anna Esbensen; Juan Fortea; Sandra Giménez; Ann-Charlotte Granholm; Laura J Hahn; Elizabeth Head; Hampus Hillerstrom; Lisa M Jacola; Matthew P Janicki; Joan M Jasien; Angela R Kamer; Raymond D Kent; Bernard Khor; Jeanne B Lawrence; Catherine Lemonnier; Amy Feldman Lewanda; William Mobley; Paul E Moore; Linda Pollak Nelson; Nicolas M Oreskovic; Ricardo S Osorio; David Patterson; Sonja A Rasmussen; Roger H Reeves; Nancy Roizen; Stephanie Santoro; Stephanie L Sherman; Nasreen Talib; Ignacio E Tapia; Kyle M Walsh; Steven F Warren; A Nicole White; Guang William Wong; John S Yi
Journal:  Transl Sci Rare Dis       Date:  2021-04-15
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