DOI:
10.1055/s-00000034
Klinische Pädiatrie
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References
Vockley J, Burton B, Berry GT.
Results from a 78-week, single-arm, open-label phase 2 study to evaluate UX007 in pediatric and adult patients with severe long-chain fattyacid oxidation disorders (LC-FAOD).
J Inherit Metab Dis 2019;
42: 169-177
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