A Subcutaneously Administered Investigational RNAi Therapeutic (ALN-CC5) Targeting Complement C5 for Treatment of PNH and Complement-Mediated Diseases: Preliminary Phase 1/2 Study Results in Patients with PNH

被引：24

作者：

Hill, Anita ^{[1
]}

Valls, Anna Gaya ^{[2
]}

Griffin, Morag ^{[1
]}

Munir, Talha ^{[3
]}

Borodovsky, Anna ^{[4
]}

Kawahata, Nori ^{[4
]}

Mclean, Helen ^{[4
]}

Shi, Kelvin ^{[4
]}

Partisano, Angela M. ^{[4
]}

Kim, Jae ^{[4
]}

Najafian, Nader ^{[4
]}

Urbano, Alvaro ^{[5
]}

机构：

[1] Leeds Teaching Hosp, St James Inst Oncol, Leeds, W Yorkshire, England

[2] Hosp Clin Barcelona, Hematol Dept, Barcelona, Spain

[3] St Jamess Inst Oncol, Leeds, W Yorkshire, England

[4] Alnylam Pharmaceut, Cambridge, MA USA

[5] Univ Barcelona, Hosp Clin Barcelona, Hematol Dept, Inst Hematol & Oncol, Barcelona, Spain

来源：

关键词：

D O I：

10.1182/blood.V128.22.3891.3891

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

3891

引用

页数：5