Denali Therapeutics Says FDA Has Selected DNL126 For Participation In Support For Clinical Trials Advancing Rare Disease Therapeutics Pilot Program
Denali Therapeutics Says FDA Has Selected DNL126 For Participation In Support For Clinical Trials Advancing Rare Disease Therapeutics Pilot Program
denali therapeutics表示FDA已選擇DNL126參與壓力位，用於推進罕見疾病治療方案的臨牀試驗支持計劃。
DNL126 is an investigational enzyme replacement therapy designed to cross the BBB for the potential treatment of MPS IIIA (Sanfilippo syndrome type A).
DNL126是一種實驗性的酶替代治療藥物，旨在通過血腦屏障治療MPS IIIA（三號橋漢氏綜合症A型）可能性。
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