Brogidirsen, an investigational exon 44 skipping agent for the treatment of Duchenne muscular dystrophy: Clinical trial design (Phase 2)
Paula R. Clemens1,2, Michelle L. Previtera3 , Robert A. Crozier3 , Leslie Magnus 3 , Eric P. Hoffman4 , Hirofumi Komaki 5,6 , Yoshitsugu Aoki 7 , and Vamshi K. Rao8 1
Department of Neurology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; 2Department of Veterans Affairs Medical Center, Pittsburgh, PA, USA; 3NS Pharma, Inc., Paramus, NJ, USA; 4School of Pharmacy and Pharmaceutical Sciences, Binghamton University – SUNY, Binghamton, NY, USA; 5Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan; 6Translational Medical Center, National Center of Neurology and Psychiatry, Tokyo, Japan; 7Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan; 8Division of Neurology, Ann and Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, USA
