RT Journal Article
SR Electronic
T1 Exon-Skipping Therapy: A Roadblock, Detour, or Bump in the Road?
JF Science Translational Medicine
FD American Association for the Advancement of Science
SP 230fs14
OP 230fs14
DO 10.1126/scitranslmed.3008873
VO 6
IS 230
A1 Hoffman, Eric P.
A1 McNally, Elizabeth M.
YR 2014
UL http://stm.sciencemag.org/content/6/230/230fs14.abstract
AB Exon skipping is a promising therapeutic for Duchenne muscular dystrophy patients, but the road to drug approvals is foggy and may require more early-stage derisking and regulatory guidance.