title: The third international meeting on genetic disorders in the RAS/MAPK pathway: towards a therapeutic approach.
abstract: "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.
authors.affiliation: University of Alabama at Birmingham, Alabama.
authors.forename: Bruce
authors.initials: B
authors.lastname: Korf
authors.affiliation: Heinrich-Heine University, Düsseldorf, Germany.
authors.forename: Reza
authors.initials: R
authors.lastname: Ahmadian
authors.affiliation: (Retired) Children's Hospital of Eastern Ontario, Ottawa, Canada.
authors.forename: Judith
authors.initials: J
authors.lastname: Allanson
authors.affiliation: Tohoku University School of Medicine, Sendai, Japan.
authors.forename: Yoko
authors.initials: Y
authors.lastname: Aoki
authors.affiliation: Children's Tumor Foundation, New York, New York.
authors.forename: Annette
authors.initials: A
authors.lastname: Bakker
authors.affiliation: Manchester Centre for Genomic Medicine, UK.|Manchester Academic Health Science Center, Manchester, UK.
authors.forename: Emma Burkitt
authors.initials: EB
authors.lastname: Wright
authors.affiliation: Parent Project Muscular Dystrophy, Hackensack, New Jersey.
authors.forename: Brian
authors.initials: B
authors.lastname: Denger
authors.affiliation: Erasmus University, Netherlands.
authors.forename: Ype
authors.initials: Y
authors.lastname: Elgersma
authors.affiliation: Icahn School of Medicine at Mount Sinai, New York.
authors.forename: Bruce D
authors.initials: BD
authors.lastname: Gelb
authors.affiliation: A.I. duPont Hospital for Children, Wilmington, Delaware.
authors.forename: Karen W
authors.initials: KW
authors.lastname: Gripp
authors.affiliation: Manchester Centre for Genomic Medicine, Manchester, UK.
authors.forename: Bronwyn
authors.initials: B
authors.lastname: Kerr
authors.affiliation: Harvard Medical School and Beth Israel Deaconess Medical Cancer Center, Boston, Massachusetts.
authors.forename: Maria
authors.initials: M
authors.lastname: Kontaridis
authors.affiliation: Catalan Institute of Oncology (ICO-IDIBELL), Barcelona, Spain.
authors.forename: Conxi
authors.initials: C
authors.lastname: Lazaro
authors.affiliation: Duke University School of Medicine, Durham, North Carolina.
authors.forename: Corinne
authors.initials: C
authors.lastname: Linardic
authors.affiliation: UC California, Davis, California.
authors.forename: Reymundo
authors.initials: R
authors.lastname: Lozano
authors.affiliation: Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts.
authors.forename: Calum A
authors.initials: CA
authors.lastname: MacRae
authors.affiliation: University of Alabama at Birmingham, Alabama.
authors.forename: Ludwine
authors.initials: L
authors.lastname: Messiaen
authors.affiliation: Icahn School of Medicine at Mount Sinai, New York.
authors.forename: Sonia
authors.initials: S
authors.lastname: Mulero-Navarro
authors.affiliation: Princess Margaret Cancer Centre, University Health Network, Toronto, Ontario, Canada.
authors.forename: Benjamin
authors.initials: B
authors.lastname: Neel
authors.affiliation: Massachusetts General Hospital, Boston, Massachusetts.
authors.forename: Scott
authors.initials: S
authors.lastname: Plotkin
authors.affiliation: University of California, Davis MIND Institute, Sacramento, California.
authors.forename: Katherine A
authors.initials: KA
authors.lastname: Rauen
authors.affiliation: Boston Children's Hospital, Massachusetts.|Harvard Medical School, Massachusetts.
authors.forename: Amy
authors.initials: A
authors.lastname: Roberts
authors.affiliation: University of California, Los Angeles, California.
authors.forename: Alcino J
authors.initials: AJ
authors.lastname: Silva
authors.affiliation: National Center for Advancing Translational Sciences (NCATS), National Institutes of Health, Maryland.
authors.forename: Sitta G
authors.initials: SG
authors.lastname: Sittampalam
authors.affiliation: Plexxikon, Inc., Berkeley, California.
authors.forename: Chao
authors.initials: C
authors.lastname: Zhang
authors.affiliation: RASopathies Network USA.
authors.forename: Lisa
authors.initials: L
authors.lastname: Schoyer
mesh_terms: D030342:Genetic Diseases, Inborn / Q000235:genetics* / Q000378:metabolism; D006801:Humans; D020935:MAP Kinase Signaling System; D018631:ras Proteins / Q000378:metabolism*
chemical_list: D018631:ras Proteins
keywords: Cardio-facio-cutaneous syndrome; Costello syndrome; Noonan syndrome; Ras; neurofibromatosis