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

Publication ,  Journal Article
Hart, SJ; Visootsak, J; Tamburri, P; Phuong, P; Baumer, N; Hernandez, M-C; Skotko, BG; Ochoa-Lubinoff, C; Liogier D'Ardhuy, X; Kishnani, PS ...
Published in: Am J Med Genet A
November 2017

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.

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Published In

Am J Med Genet A

DOI

EISSN

1552-4833

Publication Date

November 2017

Volume

173

Issue

11

Start / End Page

3029 / 3041

Location

United States

Related Subject Headings

  • Mice
  • Humans
  • Down Syndrome
  • Cognitive Dysfunction
  • Cognition
  • Animals
  • 3202 Clinical sciences
  • 3105 Genetics
  • 1103 Clinical Sciences
  • 0604 Genetics
 

Citation

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ICMJE
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Hart, S. J., Visootsak, J., Tamburri, P., Phuong, P., Baumer, N., Hernandez, M.-C., … Spiridigliozzi, G. A. (2017). Pharmacological interventions to improve cognition and adaptive functioning in Down syndrome: Strides to date. Am J Med Genet A, 173(11), 3029–3041. https://doi.org/10.1002/ajmg.a.38465
Hart, Sarah J., Jeannie Visootsak, Paul Tamburri, Patrick Phuong, Nicole Baumer, Maria-Clemencia Hernandez, Brian G. Skotko, et al. “Pharmacological interventions to improve cognition and adaptive functioning in Down syndrome: Strides to date.Am J Med Genet A 173, no. 11 (November 2017): 3029–41. https://doi.org/10.1002/ajmg.a.38465.
Hart SJ, Visootsak J, Tamburri P, Phuong P, Baumer N, Hernandez M-C, et al. Pharmacological interventions to improve cognition and adaptive functioning in Down syndrome: Strides to date. Am J Med Genet A. 2017 Nov;173(11):3029–41.
Hart, Sarah J., et al. “Pharmacological interventions to improve cognition and adaptive functioning in Down syndrome: Strides to date.Am J Med Genet A, vol. 173, no. 11, Nov. 2017, pp. 3029–41. Pubmed, doi:10.1002/ajmg.a.38465.
Hart SJ, Visootsak J, Tamburri P, Phuong P, Baumer N, Hernandez M-C, Skotko BG, Ochoa-Lubinoff C, Liogier D’Ardhuy X, Kishnani PS, Spiridigliozzi GA. Pharmacological interventions to improve cognition and adaptive functioning in Down syndrome: Strides to date. Am J Med Genet A. 2017 Nov;173(11):3029–3041.
Journal cover image

Published In

Am J Med Genet A

DOI

EISSN

1552-4833

Publication Date

November 2017

Volume

173

Issue

11

Start / End Page

3029 / 3041

Location

United States

Related Subject Headings

  • Mice
  • Humans
  • Down Syndrome
  • Cognitive Dysfunction
  • Cognition
  • Animals
  • 3202 Clinical sciences
  • 3105 Genetics
  • 1103 Clinical Sciences
  • 0604 Genetics