在针对性药物问世前，治疗手段十分有限且充满挑战。外科肢体延长术创伤性强、风险高，且行业内尚未形成统一诊疗共识；重组人生长激素（rhGH）虽能在一定程度上改善生长状况，但疗效有限，治疗2年后骨骼生长的有益作用便会减弱，还存在显著的长期副作用。
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