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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