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Ultragenyx unwraps data that may be central to its gene therapy resubmission
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1 Articles
Ultragenyx unwraps data that may be central to its gene therapy resubmission
Ultragenyx outlined new data on Tuesday that it hopes will convince the FDA to approve its recently resubmitted rare disease gene therapy. Among 17 patients with Sanfilippo syndrome type A who received the therapy, cognition ...
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