Dr. Jerry Mendell, Director of the Centers for Gene Therapy and Muscular Dystrophy at Nationwide Children's Hospital and principal investigator of the Phase IIb eteplirsen study, presented the full set of 48-week data from Sarepta at the World Muscle Society (WMS) Congress in Perth, Australia on Friday, October 13. This presentation  includes greater detail around the full Phase IIb study results.

Additional data presented at WMS included:

• Individual patient data on the primary endpoint of change in dystrophin-positive fibers from baseline;
• Additional biochemical findings including RT-PCR and western blot images from selected patients;
• Additional information on the two patients in the 30 mg/kg cohort who showed a rapidly progressive decline on the 6-minute walk test and were excluded from the analysis; and
• A summary of treatment-emergent adverse events comparing eteplirsen-treated patients versus placebo, which demonstrated that eteplirsen was well-tolerated through 48 weeks of treatment. No treatment-related adverse events, serious adverse events, or treatment discontinuations related to eteplirsen were observed.  In addition, no treatment-related changes were detected on any safety laboratory parameters, including several biomarkers for renal function.

We look forward to the Sarepta webinar on October 23, where your questions will be answered by the Sarepta team. Learn more about the upcoming webinar.


Learn more

• Read the full presentation of 48-week data from Dr. Mendell
• Read Holly Peay’s blog about what this data may mean for the community.
• Read Sharon Hesterlee’s blog about what exon skipping success means for those with Duchenne unaffected by exon skipping.