[4] Ultragenyx collaborates on product development with other companies including GeneTX, Kyowa Hakko Kirin, Mereo Biopharma and Daiichi Sankyo.

[5] Ultragenyx has three products Burosumab, Triheptanoin and Vestronidase alfa that have received FDA approval and several others currently in clinical trials.

The company also holds the non-US commercial rights to Regeneron’s evinacumab-dgnb, which is approved by the FDA and EMA.

[17] In 2017, the company discontinued development of their Ace-ERs treatment for GNE myopathy after the product did not meet the study's primary endpoint, which was significant improvement in arm strength.

It also failed to meet three secondary endpoints that evaluated patients' physical functions: leg muscle strength and knee extension force,[18] which previous trials in mice had shown efficacy.