What Can We Learn From Clinical Trials of Exon Skipping for DMD?

• Qi-long Lu,
• Sebahattin Cirak,
• Terence Partridge

Affiliations

Qi-long Lu

Department of Neurology, McColl Lockwood Laboratory for Muscular Dystrophy Research, Carolinas Medical Center, Charlotte, North Carolina, USA

Sebahattin Cirak

Center for Genetic Medicine, Children's National Medical Center, NW, Washington, DC, USA

Terence Partridge

Center for Genetic Medicine, Children's National Medical Center, NW, Washington, DC, USA