Exon-skipping therapy: a roadblock, detour, or bump in the road?
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.
Center for Genetic Medicine Research, Children's National Medical Center, 111 Michigan Avenue, NW, Washington, DC 20010, USA.
Eric P
EP
Hoffman
Elizabeth M
EM
McNally
D002986:Clinical Trials as Topic; D016189:Dystrophin / Q000235:genetics / Q000627:therapeutic use; D005091:Exons / Q000235:genetics*; D015316:Genetic Therapy; D006801:Humans; D009136:Muscular Dystrophies / Q000235:genetics / Q000628:therapy
D016189:Dystrophin