Late breaking news e-poster presentation

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).

Source: Neuromuscular Disorders - September 28, 2020 Category: Neurology Authors: Dr. Rebecca J Willcocks, Dr. Sean C Forbes, Dr. Donovan J Lott, Dr. Claudia R Senesac, Dr. Alison M Barnard, Kelly J Lehman, Carrie E Nease, Kathleen Church, Dr. Zarife Sahenk, Dr. H. Lee Sweeney, Dr. Louise R Rodino-Klapac, Dr. Glenn A Walter, Dr. Jerry Source Type: research