The third international meeting on genetic disorders in the RAS/MAPK pathway: towards a therapeutic approach.

Journal Article

"The Third International Meeting on Genetic Disorders in the RAS/MAPK Pathway: Towards a Therapeutic Approach" was held at the Renaissance Orlando at SeaWorld Hotel (August 2-4, 2013). Seventy-one physicians and scientists attended the meeting, and parallel meetings were held by patient advocacy groups (CFC International, Costello Syndrome Family Network, NF Network and Noonan Syndrome Foundation). Parent and patient advocates opened the meeting with a panel discussion to set the stage regarding their hopes and expectations for therapeutic advances. In keeping with the theme on therapeutic development, the sessions followed a progression from description of the phenotype and definition of therapeutic endpoints, to definition of genomic changes, to identification of therapeutic targets in the RAS/MAPK pathway, to preclinical drug development and testing, to clinical trials. These proceedings will review the major points of discussion.

Full Text

Duke Authors

Cited Authors

  • Korf, B; Ahmadian, R; Allanson, J; Aoki, Y; Bakker, A; Wright, EB; Denger, B; Elgersma, Y; Gelb, BD; Gripp, KW; Kerr, B; Kontaridis, M; Lazaro, C; Linardic, C; Lozano, R; MacRae, CA; Messiaen, L; Mulero-Navarro, S; Neel, B; Plotkin, S; Rauen, KA; Roberts, A; Silva, AJ; Sittampalam, SG; Zhang, C; Schoyer, L

Published Date

  • August 2015

Published In

Volume / Issue

  • 167A / 8

Start / End Page

  • 1741 - 1746

PubMed ID

  • 25900621

Pubmed Central ID

  • PMC4515140

Electronic International Standard Serial Number (EISSN)

  • 1552-4833

Digital Object Identifier (DOI)

  • 10.1002/ajmg.a.37089


  • eng

Conference Location

  • United States