Ultragenyx stumbles in P3 trial for severe muscle disease

04:40 EDT 23 Aug 2017 | Pharmafile

Ultragenyx has revealed results from a Phase 3 trial into the treatment of GNE myopathy showed that its compound did not successfully meet the primary or secondary endpoints of the study.

The failure in the clinic means that the biotech now plans to abandon all development of the drug and open up “valuable natural history data and development tools for the development of other therapies” to investigators and patient groups.

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Original Article: Ultragenyx stumbles in P3 trial for severe muscle disease


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