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PubPharm (202)
1
Clonal hematopoiesis is driven by aberrant activation of TCL1A
enthalten in:
bioRxiv.org
| 2024
von
Weinstock, J.
|
Gopakumar, J.
|
Burugula, B.
| +103
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2
Genetic control of mRNA splicing as a potential mechanism for incomplete penetrance of rare coding variants
enthalten in:
bioRxiv.org
| 2024
von
Einson, J.
|
Glinos, D.
|
Boerwinkle, E.
| +30
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3
The importance and evolution of bleeding disorder registries
enthalten in:
Haemophilia : the official journal of the World Federation of Hemophilia
| 2024
von
Tran, H.
|
Yang, R.
|
Fischer, K.
| +2
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4
Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B) : 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial
enthalten in:
The Lancet. Haematology
| 2024
von
Coppens, M.
|
Pipe, S.
|
Miesbach, W.
| +36
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5
Giroctocogene fitelparvovec gene therapy for severe hemophilia A : 104-week analysis of the phase 1/2 Alta study
enthalten in:
Blood
| 2024
von
Leavitt, A.
|
Konkle, B.
|
Stine, K.
| +15
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6
Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia
enthalten in:
Gene therapy
| 2024
von
Pabinger, I.
|
Ayash-Rashkovsky, M.
|
Escobar, M.
| +8
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7
A global comparative field study to evaluate the factor VIII activity of efanesoctocog alfa by one-stage clotting and chromogenic substrate assays at clinical haemostasis laboratories
enthalten in:
Haemophilia : the official journal of the World Federation of Hemophilia
| 2024
von
Pipe, S.
|
Sadeghi-Khomami, A.
|
Konkle, B.
| +8
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8
Landmark endorsement of a global registry : The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP), publicly endorses World Federation of Hemophilia Gene Therapy Registry as global standard
enthalten in:
Haemophilia : the official journal of the World Federation of Hemophilia
| 2024
von
Konkle, B.
|
Peyvandi, F.
|
Coffin, D.
| +4
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9
The importance and evolution of bleeding disorder registries
enthalten in:
Haemophilia
| 2024
von
Tran, H.
|
Yang, R.
|
Fischer, K.
| +2
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10
Dynamic and functional linkage between von Willebrand factor and ADAMTS-13 with aging : an Atherosclerosis Risk in Community study
enthalten in:
Journal of thrombosis and haemostasis : JTH
| 2023
von
Liu, W.
|
Patel, K.
|
Wang, Y.
| +10
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Blood
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Haemophilia : the official journal of the World...
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Transfusion
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International journal of hematology
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