16 Apr 2015 → 17 Apr 2015 Symposium Novel Therapies for Monogenic Diseases 16 Apr 2015 → 17 Apr 2015 Share Facebook X (ex-Twitter) Linkedin Copy url
from Thursday 16 April to Friday 17 April 2015 See also: Alain FischerJean-Louis Mandel Documents and media Download program pdf (218.63 KB) Program Symposium 09:30 - 10:15 Not recorded Genome Engineering with CRISPR-Cas9: Biology, Mechanisms and Applications in Medicine Emmanuelle Charpentier 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 09:30 - 10:15 Symposium 10:15 - 11:00 Not recorded Gene Therapy of Primary Immunodeficiencies (PID) Alain Fischer 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 10:15 - 11:00 Symposium 11:00 - 11:45 Not recorded Genetic Engineering of Human Hematopoiesis for the Treatment of Inherited Diseases and Cancer Luigi Naldini 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 11:00 - 11:45 Symposium 14:15 - 15:00 Not recorded Gene Therapy of Hemophilias Amit Nathwani 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 14:15 - 15:00 Symposium 15:00 - 15:35 Not recorded New Mutation-Specific Pharmacotherapies for Cystic Fibrosis Kris de Boeck 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 15:00 - 15:35 Symposium 15:35 - 15:55 Not recorded Added Value of New Genomics Technologies in Undiagnosed Patients Who May Benefit from Known Therapies Judith Melki 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 15:35 - 15:55 Symposium 15:55 - 16:30 Not recorded From Promising Clinical Studies to Recent Clinical Trials for the Fragile X Mental Retardation Syndrome: The Challenge of Judging Efficacy Vincent des Portes 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 15:55 - 16:30 Symposium 17:00 - 17:35 Not recorded The Discovery and Development of Olesoxime for Spinal Muscular Atrophy Therapy Rebecca Pruss 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 17:00 - 17:35 Symposium 17:35 - 17:55 Not recorded From Gene Identification to Functional Analysis and Treatment of Achondroplasia Laurence Legeai-Mallet 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 17:35 - 17:55 Symposium 17:55 - 18:15 Not recorded Inhibition of Thromboxane Synthase, a New Mechanism to Increase Bone Density? Valerie Cormier-Daire 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 17:55 - 18:15 Symposium 18:15 - 18:30 Not recorded Selected Oral Communications: Drug Screening on Hutchinson Gilford Progeria Using Pluripotent Stem Cells Reveals New Pharmacological Modulators of Prelamin a Farnesylation Xavier Nissan 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 18:15 - 18:30 Symposium 09:00 - 09:45 Not recorded Novel Approaches for Treatment of Retinal Diseases José-Alain Sahel 17 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 17 Apr 2015 09:00 - 09:45 Symposium 09:45 - 10:15 Not recorded Gene Replacement Therapy for Myotubular Myopathy Anna Buj Bello 17 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 17 Apr 2015 09:45 - 10:15 Symposium 10:15 - 10:35 Not recorded REPS1 Is a Novel Gene of Neurodegeneration with Brain Iron Accumulation Arnold Munnich 17 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 17 Apr 2015 10:15 - 10:35 Symposium 11:00 - 11:30 Not recorded Efficient AAV Gene Therapy in Cardiac and Neurological Murine Models for Friedreich Ataxia Hélène Puccio 17 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 17 Apr 2015 11:00 - 11:30 Symposium 11:30 - 11:45 Not recorded Selected Oral Communication: High Expression and Therapeutic Efficiency of Systemic Delivery of a Dual AAV Strategy in a Murine Model for Dysferlin Deficiencies William Lostal 17 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 17 Apr 2015 11:30 - 11:45 Symposium 11:45 - 12:00 Not recorded Selected Oral Communication: Gene Therapy for Alzheimer's Disease: The Cholesterol Connection Nathalie Cartier 17 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 17 Apr 2015 11:45 - 12:00 Symposium 12:00 - 12:45 Not recorded Round Table: Can We Afford Cost of Innovative Therapies for Rare Diseases? Franck Dufour et Serge Braun 17 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 17 Apr 2015 12:00 - 12:45 See also Alain Fischer, chair Experimental medicine Jean-Louis Mandel, chair Human genetics
Symposium 09:30 - 10:15 Not recorded Genome Engineering with CRISPR-Cas9: Biology, Mechanisms and Applications in Medicine Emmanuelle Charpentier 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 09:30 - 10:15
Symposium 10:15 - 11:00 Not recorded Gene Therapy of Primary Immunodeficiencies (PID) Alain Fischer 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 10:15 - 11:00
Symposium 11:00 - 11:45 Not recorded Genetic Engineering of Human Hematopoiesis for the Treatment of Inherited Diseases and Cancer Luigi Naldini 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 11:00 - 11:45
Symposium 14:15 - 15:00 Not recorded Gene Therapy of Hemophilias Amit Nathwani 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 14:15 - 15:00
Symposium 15:00 - 15:35 Not recorded New Mutation-Specific Pharmacotherapies for Cystic Fibrosis Kris de Boeck 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 15:00 - 15:35
Symposium 15:35 - 15:55 Not recorded Added Value of New Genomics Technologies in Undiagnosed Patients Who May Benefit from Known Therapies Judith Melki 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 15:35 - 15:55
Symposium 15:55 - 16:30 Not recorded From Promising Clinical Studies to Recent Clinical Trials for the Fragile X Mental Retardation Syndrome: The Challenge of Judging Efficacy Vincent des Portes 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 15:55 - 16:30
Symposium 17:00 - 17:35 Not recorded The Discovery and Development of Olesoxime for Spinal Muscular Atrophy Therapy Rebecca Pruss 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 17:00 - 17:35
Symposium 17:35 - 17:55 Not recorded From Gene Identification to Functional Analysis and Treatment of Achondroplasia Laurence Legeai-Mallet 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 17:35 - 17:55
Symposium 17:55 - 18:15 Not recorded Inhibition of Thromboxane Synthase, a New Mechanism to Increase Bone Density? Valerie Cormier-Daire 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 17:55 - 18:15
Symposium 18:15 - 18:30 Not recorded Selected Oral Communications: Drug Screening on Hutchinson Gilford Progeria Using Pluripotent Stem Cells Reveals New Pharmacological Modulators of Prelamin a Farnesylation Xavier Nissan 16 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 16 Apr 2015 18:15 - 18:30
Symposium 09:00 - 09:45 Not recorded Novel Approaches for Treatment of Retinal Diseases José-Alain Sahel 17 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 17 Apr 2015 09:00 - 09:45
Symposium 09:45 - 10:15 Not recorded Gene Replacement Therapy for Myotubular Myopathy Anna Buj Bello 17 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 17 Apr 2015 09:45 - 10:15
Symposium 10:15 - 10:35 Not recorded REPS1 Is a Novel Gene of Neurodegeneration with Brain Iron Accumulation Arnold Munnich 17 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 17 Apr 2015 10:15 - 10:35
Symposium 11:00 - 11:30 Not recorded Efficient AAV Gene Therapy in Cardiac and Neurological Murine Models for Friedreich Ataxia Hélène Puccio 17 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 17 Apr 2015 11:00 - 11:30
Symposium 11:30 - 11:45 Not recorded Selected Oral Communication: High Expression and Therapeutic Efficiency of Systemic Delivery of a Dual AAV Strategy in a Murine Model for Dysferlin Deficiencies William Lostal 17 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 17 Apr 2015 11:30 - 11:45
Symposium 11:45 - 12:00 Not recorded Selected Oral Communication: Gene Therapy for Alzheimer's Disease: The Cholesterol Connection Nathalie Cartier 17 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 17 Apr 2015 11:45 - 12:00
Symposium 12:00 - 12:45 Not recorded Round Table: Can We Afford Cost of Innovative Therapies for Rare Diseases? Franck Dufour et Serge Braun 17 Apr 2015 Novel Therapies for Monogenic Diseases Amphithéâtre Maurice Halbwachs, Site Marcelin Berthelot 17 Apr 2015 12:00 - 12:45
