Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria

Author:

Sardh Eliane1,Harper Pauline1,Balwani Manisha1,Stein Penelope1,Rees David1,Bissell D. Montgomery1,Desnick Robert1,Parker Charles1,Phillips John1,Bonkovsky Herbert L.1,Vassiliou Daphne1,Penz Craig1,Chan-Daniels Amy1,He Qiuling1,Querbes William1,Fitzgerald Kevin1,Kim Jae B.1,Garg Pushkal1,Vaishnaw Akshay1,Simon Amy R.1,Anderson Karl E.1

Affiliation:

1. From the Porphyria Centre Sweden, Karolinska Institutet, Karolinska University Hospital, Stockholm (E.S., P.H., D.V.); Icahn School of Medicine at Mount Sinai, New York (M.B., R.D.); King’s College Hospital, London (P.S., D.R.); University of California, San Francisco, San Francisco (D.M.B.); University of Utah, Salt Lake City (C. Parker, J.P.); Wake Forest University, Winston-Salem, NC (H.L.B.); Alnylam Pharmaceuticals, Cambridge, MA (C. Penz, A.C.-D., Q.H., W.Q., K.F., J.B.K., P.G., A.V., A.R.S.); and...

Funder

Alnylam Pharmaceuticals

Publisher

Massachusetts Medical Society

Subject

General Medicine

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