REGENXBIO's partnership with Nippon Shinyaku provides $110M upfront, ensuring a cash runway for gene therapy programs in MPS ...
The FDA established its accelerated approval program in 1992 to allow drugs to reach the market quickly for life-threatening ...
Sarepta Therapeutics' Elevidys for DMD shows revenue potential, with market cap justified at $11.4bn and potential for >50% ...
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