Defeat Duchenne Canada is saddened to learn that Sarepta has discontinued its MOMENTUM study of SRP-5051, an investigational peptide-conjugated PMO (PPMO; an engineered strand of DNA that is meant to change how a gene acts) in people with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. Although the early increases in dystrophin levels were encouraging, safety concerns about low blood magnesium levels that continued despite stopping treatment and negative results of kidney function assessments led to the decision to stop the clinical development program for SRP-5051.

The MOMENTUM study was a global study which included London Health Sciences Centre (LHSC) as the only Canadian study site. Sarepta has four FDA-approved treatments in the United States and other jurisdictions, including ELEVIDYS®, Exondys 51®, VYONDYS 53®, and AMONDYS 45®. See their website for more information about their currently approved treatments on their website.

Please see Sarepta’s Community Letter for additional details.

Were you or your child participating in the MOMENTUM study and need support? Please contact us to help you navigate next steps.

Want to know more about Duchenne Research?

Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: