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YAQ006 and YAQ007 are, respectively, the intravenous and oral formulations of L-ornithine phenylacetate (L-OPA), a potent ammonia-scavenging agent. Yaqrit is preparing to start a phase 3 trial ...
A decrease in VLCFA levels in brain, the main tissue affected in X-ALD, is likely because it has been shown that phenylacetate, the initial product of 4PBA breakdown, can cross the blood–brain ...