Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria.
Sardh E, Harper P, Balwani M, Stein P, Rees D, Bissell DM, Desnick R, Parker C, Phillips J, Bonkovsky HL, Vassiliou D, Penz C, Chan-Daniels A, He Q, Querbes W, Fitzgerald K, Kim JB, Garg P, Vaishnaw A, Simon AR, Anderson KE.
Sardh E, et al. Among authors: harper p.
N Engl J Med. 2019 Feb 7;380(6):549-558. doi: 10.1056/NEJMoa1807838.
N Engl J Med. 2019.
PMID: 30726693
Clinical Trial.