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Strategies & Market Trends : 2026 TeoTwawKi ... 2032 Darkest Interregnum -- Ignore unavailable to you. Want to Upgrade?

To: Julius Wong who wrote (198055)	4/9/2023 11:37:02 AM
From: THE ANT	1 Recommendation Recommended By Julius Wong Respond to of 218262

People worry that they’re gene therapy will obliviate the need for their pmo therapy but they are wrong. The missing protein in Duquesne‘s muscular dystrophy is so long that the gene therapy makes a truncated protein which is still significantly effective. The PMO puts a patch on the DNA that produces a small amount of full length protein. Thus both medication’s will be needed for optimal response. Sarepta has also been using technology from multiple new companies that will be involved in the future of gene therapy. They could’ve saved a lot of money by not doing this, but by showing the FDA they are at the forefront of gene therapy and looking to the future. They will likely form a bond with the FDA. Going forward.