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THE WALL STREET JOURNAL · GLOBAL ·

Exclusive | FDA Gives Third Rare-Disease Drug Another Shot, Regenxbio Says - WSJ

As reported by The Wall Street Journal
Company plans to refile its application for its gene therapy for a fatal brain disease after FDA dropped demands to give a placebo to some study subjects
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Our editorial commentary, not the publisher's article body.

Regenxbio is set to refile its application for a gene therapy targeting a fatal brain disease. This follows the FDA dropping its demand for a placebo group in the study. This marks the third rare-disease drug to receive a second chance from the FDA, signaling positive momentum…

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