FDA paves way for clinical trials of Sarepta’s Duchenne MD therapy SRP-5051 | DuchenneXchange

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FDA paves way for clinical trials of Sarepta’s Duchenne MD therapy SRP-5051


The U.S. Food and Drug Administration has taken a step that will allow Sarepta Therapeutics to start clinical trials of its Duchenne muscular dystrophy therapy SRP-5051.

That move was accepting the company’s Investigational New Drug application for SRP-5051. The therapy, known as a peptide phosphorodiamidate morpholino oligomer, is designed to treat those whose Duchenne MD stems from the exon 51 genetic mutation. They constitute the largest group of DMD patients — about 13 percent.

Genes are made up of dozens of components known as exons. A missing exon can lead to a gene mutation that generates a faulty protein, causing a disease.

 

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