Assessment of rAAVrh.74.MHCK7.micro-dystrophin Gene Therapy Using Magnetic Resonance Imaging in Children With Duchenne Muscular Dystrophy

JAMA Netw Open. 2021 Jan 4;4(1):e2031851. doi: 10.1001/jamanetworkopen.2020.31851.

Abstract

This case-control study uses magnetic resonance imaging and spectroscopy to evaluate the association between treatment with recombinant adeno-associated virus serotype rh74 (rAAVrh74) and muscle quality in children with Duchenne muscular dystrophy.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Case-Control Studies
  • Child
  • Child, Preschool
  • Dystrophin / genetics*
  • Genetic Therapy / methods*
  • Humans
  • Magnetic Resonance Imaging / methods*
  • Muscle, Skeletal / diagnostic imaging
  • Muscular Dystrophy, Duchenne / diagnostic imaging*
  • Muscular Dystrophy, Duchenne / therapy*
  • Treatment Outcome

Substances

  • Dystrophin