Sarepta Therapeutics has recently released an update from its MOMENTUM study investigating SRP-5051 for the treatment of boys with Duchenne muscular dystrophy amenable to skipping of exon 51. This is a clinical trial of a ‘next-generation’ of exon-skipping (molecular patch) drug that the company hopes will be more effective with less frequent dosing.
The update shares results from four boys who are in the cohort receiving the treatment at a higher dose (30mg/kg). The study showed that monthly dosing led to an increase in exon skipping and dystrophin protein in the muscle after 12 weeks. Some of the boys developed a drop in levels of magnesium (reported as a serious adverse event), which was treated with magnesium supplements.
Further research is needed to test if the increase in exon skipping and dystrophin protein results in any changes in muscle function, for example changes to muscle strength.
You can read more in Sarepta Therapeutics press release .
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