Gene Therapy Approaches to Replace or Restore Dystrophin

PF-06939926 – Kasia Lobello, MD (Pfizer, Inc.)
RGX-202 – Jahannaz Dastgir, DO (REGENXBIO)
scAAV9.U7-ACCA – Kevin Flanigan, MD (Nationwide Children’s Hospital)
SGT-001 – Carl Morris, PhD (Solid Biosciences)
SRP-9001 – Teji Singh, MD (Sarepta Therapeutics)