On January 7, Sarepta Therapeutics announced their topline results from their gene therapy candidate SRP-9001. This study is an ongoing, randomized, double-blinded, placebo-controlled trial evaluating the safety, efficacy and tolerability of a single dose of SRP-9001 in 41 Duchenne patients aged 4-7. The study met its biological primary endpoint of micro-dystrophin expression at 12 weeks. It did show an increase in the NSAA functional outcome at 48 weeks, but this result did not reach statistical significance. They believe the reason for this was due to an imbalance in the NSAA total score at baseline in a cohort of patients aged 6-7 years old.

To read more about the results, click here.

To read Sarepta’s community letter, click here.

Sarepta continues to remain focused on moving forward with their gene therapy and will use these results to refine their protocol for their upcoming trial.

We appreciate the commitment of all the families who have participated in this trial and will continue to follow the progress closely.

About Sarepta Therapeutics

Sarepta Therapeutics has built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Company’s programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visit www.sarepta.com.