Sarepta Therapeutics is a biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare, infectious, and other diseases.
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|Sarepta Therapeutics Announces Presentations at the 2017 MDA Scientific Conference|
Details of Sarepta’s presentations at the
Oral Presentation and Poster:
Title: Long-Term Treatment with Eteplirsen in Non-ambulatory Patients: A Case Study in Identical Twins
Title: Effects of Long-Term Treatment With Eteplirsen on Pulmonary Function in Patients With Duchenne Muscular Dystrophy: Findings of Two Phase 2 Clinical Trials
About EXONDYS 51™
EXONDYS 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. EXONDYS 51 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 EXONDYS 51 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 EXONDYS 51 has not been established.
Important Safety Information
Please see the EXONDYS 51 (eteplirsen) U.S. Full Prescribing Information at www.EXONDYS51.com.
About Sarepta Therapeutics
Internet Posting of Information
Media and Investors:
“Safe Harbor” Statement under the Private Securities Litigation Reform Act of 1995: The statements that are not historical facts contained in this release are forward–looking statements that involve risks and uncertainties, including, but not limited to, the results of research and development efforts, the results of preclinical and clinical testing, the effect of regulation by the FDA and other agencies, the impact of competitive products, product development, commercialization and technological difficulties, and other risks detailed in the company’s Securities and Exchange Commission filings.