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Sarepta Therapeutics Announces Positive Expression Results From The Casimersen (SRP-4045) Arm Of The ESSENCE Study
Clinical Trials and Research

Sarepta Therapeutics Announces Positive Expression Results from the Casimersen (SRP-4045) Arm of the ESSENCE Study

Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, today announced results from its interim analysis of muscle biopsy endpoints comparing casimersen treatment to placebo in the ESSENCE study, also known as study 4045-301. ESSENCE is a global, randomized double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of casimersen and golodirsen in patients amenable to skipping exons 45 or 53, respectively.

After soliciting feedback from the FDA, Sarepta conducted an interim analysis for levels of dystrophin protein expression in those patients who are amenable to exon 45 skipping to determine the potential for a New Drug Application (NDA) filing based on dystrophin as a surrogate endpoint. With these results, the Company intends to work toward submission of an NDA for casimersen in the middle of 2019.

Mar 28, 2019
Single CRISPR Treatment Provides Long-Term Benefits In Mice
Clinical Trials and Research

Single CRISPR Treatment Provides Long-Term Benefits in Mice

Researchers at Duke University have shown that a single systemic treatment using CRISPR genome editing technology can safely and stably correct a genetic disease -- Duchenne muscular dystrophy (DMD) -- for more than a year in mice, despite observed immune responses and alternative gene editing outcomes.

The study appears online on February 18in the journal Nature Medicine.

Feb 18, 2019