டக் இஂக்ரம் News Today : Breaking News, Live Updates & Top Stories | Vimarsana

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.

Louise rodino klapacDoug ingramBrian skorneyDrug administrationDive briefSolid biosciencesRodino klapacடக் இஂக்ரம்டைவ் சுருக்கமானதிட உயிர் அறிவியல்