TY - JOUR
T1 - Exon-skipping therapy
T2 - A roadblock, detour, or bump in the road?
AU - Hoffman, Eric P.
AU - McNally, Elizabeth M.
PY - 2014/4/2
Y1 - 2014/4/2
N2 - 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.
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.
UR - http://www.scopus.com/inward/record.url?scp=84899120609&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84899120609&partnerID=8YFLogxK
U2 - 10.1126/scitranslmed.3008873
DO - 10.1126/scitranslmed.3008873
M3 - Short survey
C2 - 24695683
AN - SCOPUS:84899120609
SN - 1946-6234
VL - 6
JO - Science translational medicine
JF - Science translational medicine
IS - 230
M1 - 230fs14
ER -