$AKTX ]Open pivotal study in pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA) with orphan and fast track approval Preparing for pivotal study in bullous pemphigoid (BP) following recent completion of successful Phase II study Positive EMA opinion on orphan designation for nomacopan for BP along with Orphan Drug designation granted by FDA in BP
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