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  • Conversation: Sarepta soars on positive Duchenne data

    • June 20, 2018 12:41 PM BST
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      Sarepta soars on positive Duchenne data

      Sarepta’s gene therapy for Duchenne muscular dystrophy has produced stellar results in the first three patients treated in a clinical trial, raising hopes that it could halt or even reverse the muscle wasting disease.
      The data were reported during Sarepta’s R&D update yesterday and – by the end of the day – shares in the US biotech had risen by a third, having been up almost 60% at one point. Analysts at Leerink said the results were a ‘home run’ and way ahead of expectations, and positions Sarepta as a “leader in this field.”
      DMD – a rare muscle-wasting disease that mostly affects boys and typically is fatal by age 30 – is caused by a defect in the gene coding for dystrophin, a protein found in muscles that is critical for movement.
      The data from the three boys – who appeared at the R&D event to rousing applause – showed that Sarepta’s AAVrh74.MHCK7 gene therapy led to the production of a truncated form of dystrophin at a level 38% of normal – which may not seem that impressive at first glance but according to clinicians could have a dramatic impact on the disease.
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