
After gene therapy deaths, Astellas to revisit fatal neuromuscular condition in partnership with biotech startup
Four years ago, a biotech company presented data so powerful it made scientists cry.
Speaking to a packed room at an annual cell and gene therapy conference in 2019, Audentes Therapeutics played a video of a young boy who could scarcely move and required a ventilator to breathe. Born with a fatal neuromuscular condition called X-linked myotubular myopathy, for which there are no treatments, he was out of options until Audentes came along.
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