Records containing: Trifunctional Protein (TFP) Deficiency
This is the wording recorded or indexed by the source. An agency index term is not necessarily an authorised indication. Co-listed drug names are not linked to this term as a treatment relationship.
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CLINICAL_TRIALS study record update · 2021-12-08An Open-label Long-Term Safety and Efficacy Extension Study in Subjects With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Previously Enrolled in UX007 or Triheptanoin Studies
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CLINICAL_TRIALS study record update · 2021-02-11An Open-label Phase 2 Study to Assess Safety and Clinical Effects of UX007 in Subjects With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)