A phase II double-blind multicentre, placebo-controlled trial to assess the efficacy and safety of alpelisib (BYL719) in paediatric and adult patients with Megalencephaly-CApillary... by Jean-Marc Treluyer, Benedicte Demeer, Laurence Faivre, Marc Bardou, Nathalie Boddaert, Paul Kuentz, Florence Petit, Christine Francannet, Pierre Vabres, Nadia Bahi-Buisson, Estelle Colin, Laurent Guibaud, Romaric Loffroy, Maxime Luu, Caroline Racine, Aurore Garde, Philippe Khau Van Kien, Aurore Curie, Nawale Hadouiri, Aurélie Espitalier, Agnès Maurer, Maud Carpentier, Adélaide Rega, Mouna Chebbi, Julie Charligny, Guillaume Canaud, Camille Fleck, Amelie Cransac, Elise Boucher-Brischoux, Adélaïde Brosseau-Beauvir, Florian Cherik, Alice Phan, Michaela Semeraro, Marion Nys, Charles Joris Roux, Alinoë Lavillaureix, Isabelle Maruani

Get full text

P181: Prenatal-onset hypertrophic cardiomyopathy in 54 patients with RASopathies: Understanding phenotype-genotype correlations for risk stratification, medical management and targ... by Guillaume Jouret, Dominik Brado, Jeroen Breckpot, Davide Vecchio, Mélanie Fradin, Irene Valenzuala, Paula Fernandez, Nuria Martinez, Fabio Sirchia, Alessandro Mussa, Federico Fornari, Jacqueline Aziza, Maria Isis Atallah Gonzalez, Fanny Laffargue, Gueneret Michele, Adela Chirita, Mathilde Nizon, Philippe Khau Van Kien, Francesca Mari, Anna Carrer, Maria Haanpaa, Caroline Rooryck-Thambo, Claire Beneteau, Jean-Baptiste Ichtertz, Marie Bournez, Juliette Dupont, Atilano Carcavilla, Lucia Deiros, Emikarina Rikeros, Maria De Los Angeles Gomez, Maude Grelet, Laetitia Lambert, Lucas Hérissant, Viorica Ciorna, Arthur Sorlin, Rodolphe Dard, Abdelkader Heddar, Jean-Paul Hermand, Didier Menzies

Get full text