Thérapies à l'Horizon

Thursday, March 28, 2019
28-AM-Plenary 9:00 AM > 10:30 AM Thérapies à l'Horizon Amphi A

9:00 AM S15-01 Nucleic acid-based therapies for neuromuscular disease > M. Matthew WOOD
Objectives : Oligonucleotide therapies for neuromuscular disease
Gene based therapeutic interventions have the potential to transform the treatment landscape for neuromuscular diseases. After more than a decade of effort, significant progress has been made in the development of first generation oligonucleotide therapies for Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA). These oligonucleotide drugs modulate target gene splicing in order to correct the underlying genetic defect and restore expression of essential proteins. First generation oligonucleotides, while demonstrating proof-of-principle, suffer from the limitation of inadequate tissue and cell delivery - the major barrier to the development of potent, disease modifying oligonucleotide interventions. The goal of second generation oligonucleotides is to overcome the barrier of intracellular delivery either by the development of novel oligonucleotide backbone chemistries with improved delivery properties and/or by exploitation of delivery technologies which may be coupled with oligonucleotide drugs.
9:30 AM S15-02 Tamoxifen as a treatment for muscular diseases: an unexpected facet of a repurposed anticancer drug > O. Olivier DORCHIES 10:00 AM S15-03 Vamorolone retains efficacy and reduces safety concerns of glucocorticoid drugs > E. Eric HOFFMAN

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