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After the introduction of the New Generation Sequencing (NGS) technology [30], which relies on platforms for high-throughput, massively parallel sequencing that can analyse many genes at once, it was possible to identify the forms of PFIC, from PFIC1 to PFIC13 [4, 8, 11, 33,34,35,36,37]

There is one open-label intravenous safety pilot in two healthy adults, who received single doses of 10 mg and 20 mg with no comparator, no blinding, and short-term observation limited to vital signs, self reported symptoms and labs across two days [4]