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Sarepta Therapeutics to Present at the 22nd International Annual Congress of the World Muscle Society
09/28/17 8:30 AM EDT
Dr. Muntoni is a Pediatric Neurologist,
Details of Sarepta’s other four poster presentations and its joint poster with
Title: Optimization and Implementation of Best Practices for Collection and Preparation of Muscle Biopsies for Analysis During Clinical Trials of Neuromuscular Disease Therapeutics
Date and Time:
Title: Edasalonexent (CAT-1004), an NF-kB Inhibitor, Enhances Myotube Formation In Vitro, and Increases Exon-Skipped Sarcolemmal Dystrophin in Muscle of Mdx Mice
Date and Time:
Title: Development of Novel Observer-Reported Outcome Assessments in Clinical Trials of Patients with Duchenne Muscular Dystrophy
Date and Time:
Title: Effects of Long-Term Treatment with Eteplirsen on Cardiac Function
Date and Time:
Title: Effects of Long-Term Eteplirsen Treatment on Upper Limb Function in Patients With Duchenne Muscular Dystrophy: Findings of Two Phase 2 Clinical Trials
Date and Time:
About Eteplirsen
Eteplirsen uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. Eteplirsen is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. Data from clinical studies of eteplirsen in a small number of DMD patients have demonstrated a consistent safety and tolerability profile. The pivotal trials were not designed to evaluate long-term safety and a clinical benefit of eteplirsen has not been established.
Important Safety InformationAbout Eteplirsen
Adverse reactions in DMD patients (N=8) treated with eteplirsen 30 or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (eteplirsen, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of eteplirsen is not recommended.
In the 88 patients who received ≥30 mg/kg/week of eteplirsen for up to 208 weeks in clinical studies, the following events were reported in ≥10% of patients and occurred more frequently than on the same dose in Study 1: vomiting, contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection.
There have been reports of transient erythema, facial flushing, and elevated temperature occurring on the day of eteplirsen infusion.
For further information, please see the full Prescribing Information.
About Sarepta Therapeutics
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Source: Sarepta Therapeutics
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