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Critical limitations in the current evidence include: Most studies focus on surrogate markers (liver fat content, enzyme levels) rather than hard clinical outcomes (cirrhosis prevention, liver-related mortality) Long-term safety data beyond 12 years are generally unavailable Optimal patient selection criteria remain unclear Cost-effectiveness has not been established for liver-specific indications Patients should understand that promising research findings do not constitute proof of clinical benefit or justify off-label use outside properly conducted clinical trials or specialist supervision
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Health reporter Katherine Ward has more from doctors about the rare condition, and what people taking the drug should know
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