EMERGENE Study (SRP-9003, Study 301) Community Webinar

The Speak Foundation, a patient-led organization focused on LGMDs, hosted a community webinar on Sarepta’s EMERGENE study (SRP-9003-301) on Wednesday, April 10, 2024, at 12pm ET. During the webinar, we presented an overview on:
LGMDs and the importance of knowing your subtype via genetic testing to be approved for the trial.
Clinical development of SRP-9003, Sarepta’s investigational gene therapy for LGMD2E/R4 (beta-sarcoglycanopathy)
Sarepta is advancing the science behind limb-girdle muscular dystrophy research. Sarepta currently has 6 LGMD programs in development: sarcoglycanopathies (LGMD2E/R4, LGMD2D/R3, LGMD2C/R5), dysferlinopathy (LGMD2B/R2), anoctaminopathy (LGMD2L/R12), and calpainopathy (LGMD2A/R1). Sarepta Therapeutics is a global biotechnology company on an urgent mission to engineer precision genetic medicine to reclaim futures otherwise impacted or cut short by rare diseases.