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Sarepta Therapeutics' DMD gene therapy shows early success

Sarepta Therapeutics' DMD gene therapy shows early success
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Sarepta's Duchenne gene therapy clears study hurdle, although questions linger

Dive Brief:
Sarepta Therapeutics on Tuesday disclosed results from a small but important study, showing a commercial-grade version of its gene therapy for Duchenne muscular dystrophy appears comparable to the product it used in earlier clinical testing.
The first 11 patients to receive the commercial version of the therapy produced an average of 55.4% of normal levels of micro dystrophin a shortened form of the protein Duchenne patients lack three months after treatment. That number was 51.7% for a similar group of 11 patients in an earlier trial. No new safety concerns were reported.
Sarepta plans to meet with the Food and Drug Administration to determine the next steps for its closely watched program, including the design of a Phase 3 study with the commercial material. But the biotech still has key questions to answer, most notably whether disappointing Phase 2 results reported in January were simply bad luck, as the company has contended. ....

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Sarepta Therapeutics (SRPT) Q1 2021 Earnings Call Transcript

Sarepta Therapeutics (SRPT) Q1 2021 Earnings Call Transcript
Motley Fool Transcribing
© The Motley Fool
Logo of jester cap with thought bubble.
Sarepta Therapeutics (NASDAQ: SRPT)
Good afternoon, ladies and gentlemen, and welcome to the Sarepta Therapeutics first-quarter 2021 earnings call. [Operator instructions] As a reminder, today s program is being recorded. I d now like to introduce your host for today s program, Mary Jenkins, senior management, investor relations. Please go ahead.
Mary Jenkins
Senior Management, Investor Relations
Thank you, Jonathan, and thank you all for joining today s call. Earlier today, we released our financial results for the first-quarter 2021. The press release is available on our website at sarepta.com, and our 10-Q was filed with the Securities and Exchange Commission earlier this afternoon. Joining us on the call today are Doug Ingram, Ian Estepan, Dallan Murray, Dr. ....

United States , Anupam Rama , Raymond James , Louise Rodino , Gena Wang , Mary Jenkins , Anvita Gupta , Louise Rodino Klapac , Alethia Young , Brian Abrahams , Ian Estepan , Salveen Richter , Doug Ingram , Gil Blum , America Merrill Lynch , Danielle Brill , Tim Lugo William Blair , Daniil Gataulin Raymond James , Kelly Girskis , Sonya Bhatia Goldman Sachs , Brian Skorney , Tim Lugo , Gilmore Oneill , Anvita Gupta Cowen , Colin Bristow , Tazeen Ahmad ,

Sarepta Therapeutics Reports Positive Clinical Results from Phase 2 MOMENTUM Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51

Home / Top News / Sarepta Therapeutics Reports Positive Clinical Results from Phase 2 MOMENTUM Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51
Sarepta Therapeutics Reports Positive Clinical Results from Phase 2 MOMENTUM Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51
Results suggest a highly potent next-generation treatment that could offer greater efficacy with less frequent dosing
SRP-5051 dosed monthly at 30 mg/kg delivered mean exon skipping of
10.79% and mean dystrophin expression of 6.55%,
consistently higher than the other SRP-5051 dosing cohorts at 12 weeks and weekly eteplirsen at 24 weeks
Sarepta’s predictive model indicates that ....

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DMD | Benzinga

Just weeks into its life as a publicly traded company, Solid Biosciences Inc (NASDAQ: SLDB) caught the eyes of four analysts who assumed coverage Tuesday with a mix of Holds and Buys.
Here is what one of the more bearish critics had to say.
The Rating
Goldman Sachs analyst Salveen Richter. Read More. ....

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