プラスミノーゲン欠乏症(PLGD)の最新の進歩は何ですか?

ここでプラスミノーゲン欠乏症(PLGD)に関する最新の進歩と発見を見ることができます。

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Prometic Life Sciences is developing a new plasminogen replacement therapy for congenital PLGD. Interim trial results from a Phase 2/3 study in patients with congenital PLGD have demonstrated that plasminogen IV effectively resolved 90.9% of visible lesions within 12 weeks and no new lesions developed during this treatment period. Non-visible (systemic) lesions also showed improvement. Plasminogen IV therapy has been shown to be well tolerated and safe. (C. Nakar, A. Shapiro et al; ISTH, 2017). Plasminogen replacement therapy has received orphan drug designation and priority review by the FDA. Official FDA review is ongoing at the time of this writing.

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