Zolgensma 結果共2筆

台中一名新生兒出生第10天，就被診斷出患有與知名人權律師陳俊翰同款的「SMA脊髓性肌肉萎縮症」，出現肌肉無力、吞嚥功能不佳等症狀，中山醫評估後為該童申請一劑要價4千9百萬元基因治療針劑「Zolgensma」，施打兩週肌肉功能和呼吸吞嚥都有改善，同時這也是該針劑納入健保給付後中區首例，也是全台最年輕治療成功案例。

The National Health Insurance (NHI) Administration in Taiwan has announced the approval of new applications for Spinal Muscular Atrophy (SMA) treatment coverage. This decision comes after the passing of lawyer Chen Chun-han, a prominent advocate for wider medication coverage for SMA. SMA is a progressive neuro-muscular degenerative disease caused by a mutation in the SMN1 gene, impacting muscle functionality and activities such as sitting, walking, speaking, breathing, and eating. The NHI Administration has included the gene therapy drug Zolgensma, which carries a price tag of NT＄49 million, in its benefits since the second half of 2023. The criteria for medication eligibility include an upper limb motor function index (RULM) score of at least 15. There are over 400 SMA patients in Taiwan, and by the end of 2023, 158 people applied for medication, with 145 approvals. The NHI Administration plans to hold an expert meeting in March to discuss expanding access to the drugs for all SMA patients, with a potential approval in the first half of 2024.