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December 7, 2012
08:34 EDTSRPTSarepta updates data from Study 202 of steplirsen for treatment of DMD
Sarepta Therapeutics announced updated data from Study 202, its open-label, Phase IIb extension study of eteplirsen for the treatment of Duchenne muscular dystrophy, or DMD. Patients treated with eteplirsen for 62 weeks and evaluable on ambulatory measures maintained a statistically significant clinical benefit on the primary clinical outcome measure, the 6-minute walk test, or 6MWT, compared to patients who received placebo for 24 weeks followed by 38 weeks of eteplirsen treatment. The mITT consisted of 10 of the enrolled 12 patients, and excludes two patients who showed signs of rapid disease progression and lost ambulation by week 24. The placebo/delayed-treatment cohort (n=4) also demonstrated stability in walking distance from week 36 through week 62 with a less than 10 meter change over this timeframe, the period in which dystrophin was likely produced, with confirmation of significant dystrophin levels at week 48 through analysis of muscle biopsies in these patients.
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August 21, 2014
09:01 EDTSRPTSarepta enters into partnership with Flagship Biosciences
Sarepta Therapeutics and Flagship Biosciences announced a multi-year, multi-product partnership for the development of automated quantitative endpoint measurements in muscular dystrophy to support the advancement of Sarepta’s Duchenne muscular dystrophy drug pipeline, including its lead candidate, eteplirsen. This newly established collaboration with Flagship demonstrates Sarepta’s commitment to enhancing the objective measurement of dystrophin in tissue samples for its growing pipeline of RNA-based therapeutics to treat DMD.

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