Expert Interview
Understanding UX007 potential in long-chain fatty acid oxidation disorder (LC-FAOD) after data released from a Phase 2 Open label study of 29 patients was reported
Ticker(s): RAREA doctor with experience treating patients with LC-FAOD. Ideally an investigator in one of the trials for UX007 who has experience with the data and treatment. An investigator from this study might be ideal: https://www.ncbi.nlm.nih.gov/pubmed/26116311
Please describe your background treating patients with LC-FAOD and experience with UX007.
Added By: joe_mccannHow accurate is the company's estimate of 2,000-3,500 patients with LC-FAOD in the US? Are these patients well identified and currently being treated / on registries or is that more of an epidemiological estimate?
Added By: joe_mccannDoes the endpoint of "Major Clinical Events" seem like the most relevant endpoint for this patient population? What is the minimal reduction you would consider clinical relevant?
Added By: joe_mccannAre there other conditions outside of LC-FAOD that might benefit from the UX007 mechanism?
Added By: joe_mccannA long term open label study was published on triheptanoin use over up to 12.5 years in patients with LC-FAOD https://www.ncbi.nlm.nih.gov/pubmed/26116311
Can you please discuss the differences between the UX007 program and this study?
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