Sarepta Therapeutics have announced new data from its trial testing SRP-9003 (previously MYO-101), a gene therapy for limb girdle muscular dystrophy (LGMD) type 2E. Three children with LGMD 2E have been dosed so far and no new safety concerns are reported. Nine months after receiving SRP-9003, all three patients have improved in functional tests. Sarepta will test a higher dose of SRP-9003 in some other patients and then choose the best dose to take forward into its next trial.
For more information, read Sarepta’s press release.