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Ultragenyx, after setbacks, nabs first gene therapy approval

BioPharma Dive

The clearance of Genglycos for a rare glycogen storage disease gives Ultragenyx a sellable voucher and momentum ahead of an eagerly anticipated study readout.

Rare Disease

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pharminent August 20, 2026 (Last updated: September 1, 2026)

BioPharma Dive

The clearance of Genglycos for a rare glycogen storage disease gives Ultragenyx a sellable voucher and momentum ahead of an eagerly anticipated study readout.

Rare Disease

Read full story

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