Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients with Inhibitors

被引：8

作者：

Pasi, K. John ^{[1
]}

Georgiev, Pencho ^{[2
]}

Mant, Tim ^{[3
]}

Lissitchkov, Toshko ^{[4
]}

Creagh, Michael Desmond ^{[5
]}

Bevan, David ^{[6
]}

Austin, Steve ^{[7
]}

Hay, Charles R. ^{[8
]}

Hegemann, Inga ^{[9
]}

Kazmi, Rashid ^{[10
]}

Chowdary, Pratima ^{[11
,12
]}

Ragni, Margaret V. ^{[13
,14
]}

Soh, Chang-Heok ^{[15
]}

Akinc, Akin ^{[15
]}

Partisano, Angela M. ^{[15
]}

Sorenson, Benny ^{[15
]}

Rangarajan, Savita ^{[16
]}

机构：

[1] Barts & London Queen Marys Sch Med & Dent, Royal London Haemophilia Ctr, London, England

[2] Univ Multiprofile Hosp Act Treatment Sveti Georgi, Plovdiv, Bulgaria

[3] Quintiles Drug Res Unit, London, England

[4] SHAT Joan Pavel, Dept Coagulat Disorders & Anemia, Sofia, Bulgaria

[5] Royal Cornwall Hosp NHS Trust, Truro, England

[6] Guys & St Thomas Hosp NHS Trust, London, England

[7] St Georges Univ Hosp NHS Fdn Trust, St Georges Haemophilia Ctr, London, England

[8] Manchester Royal Infirm, Manchester, Lancs, England

[9] Univ Hosp, Zurich, Switzerland

[10] Univ Hosp Southampton NHS Fdn Trust, Southampton, Hants, England

[11] Royal Free Hosp, KD Haemophilia Ctr, London, England

[12] Royal Free Hosp, Thrombosis Unit, London, England

[13] Univ Pittsburgh, Div Hematol Oncol, Dept Med, Med Ctr, Pittsburgh, PA USA

[14] Univ Pittsburgh, Hemophilia Ctr Western PA, Med Ctr, Pittsburgh, PA USA

[15] Alnylam Pharmaceut, Cambridge, MA USA

[16] Guys & St Thomas NHS Fdn Trust, Ctr Haemostasis Thrombosis, London, England

来源：

BLOOD | 2016年 / 128卷 / 22期

关键词：

D O I：

10.1182/blood.V128.22.1397.1397

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

1397

引用

页数：6

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