Abstract
A recent study of rAAVrh.74.MHCK7.micro-dystrophin gene therapy was well tolerated and showed substantial micro-dystrophin expression and improvements in functional outcomes post-gene transfer. Quantitative magnetic resonance imaging and spectroscopy (qMRI/MRS) data were collected in three participants from that study (aged 5-7) and were compared with natural history qMRI/MRS data from a large ongoing study (n=180; n=54 age- and corticosteroid-status matched to gene therapy treated boys and 23 age-matched unaffected controls).
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