Capricor’s Interim Data Could Offer Hope For Older DMD Patients
Although only interim data from six treated patients, Capricor’s Phase II DMD study shows improvements in upper limb performance, grip strength and respiration. Cardiac performance shows positive trend, but not yet statistical significance.
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Pfizer is forging ahead with a Phase III trial for its DMD therapy, even though Phase Ib data raised safety worries. Gene therapy rival Sarepta’s shares were up by 17% on the news.
CEO Doug Ingram talked with Scrip about Sarepta's plans to quickly build out a gene therapy pipeline, potentially launch another exon-skipping drug this year and to develop micro-dystrophin for Duchenne muscular dystrophy.
Sarepta Commits To Rapid, Thorough Pivotal Study For DMD Gene Therapy Based On Functional Improvements
The company presented encouraging data on functional assessments of four boys treated with a micro-dystrophin gene therapy at the World Muscle Society, furthering momentum around the program.