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PubPharm (296)
1
Recommendations for a minimum data set for monitoring gene therapy in hemophilia : communication from the ISTH SSC Working Group on Gene Therapy
enthalten in:
Journal of thrombosis and haemostasis : JTH
| 2024
von
Miesbach, W.
|
Konkle, B.
|
Chowdary, P.
| +9
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2
Deciphering conundrums of adeno-associated virus liver-directed gene therapy : focus on hemophilia
enthalten in:
Journal of thrombosis and haemostasis : JTH
| 2024
von
Pierce, G.
|
Fong, S.
|
Long, B.
| +1
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3
The underevaluated impacts of the therapeutic revolution of hemophilia on women and girls
enthalten in:
Journal of thrombosis and haemostasis : JTH
| 2024
von
Hermans, C.
|
Krumb, E.
|
Rotellini, D.
| +1
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4
Long-term safety and efficacy outcomes of valoctocogene roxaparvovec gene transfer up to 6 years post-treatment
enthalten in:
Haemophilia : the official journal of the World Federation of Hemophilia
| 2024
von
Symington, E.
|
Rangarajan, S.
|
Lester, W.
| +11
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5
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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6
The persistent educational digital divide and its impact on societal inequality
enthalten in:
PloS one
| 2024
von
Pierce, G.
|
Cleary, P.
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7
I FACTOR IX POLYPEPTIDES AND METHODS OF USE THEREOF
enthalten in:
Europäisches Patentamt
| 2023
von
PIERCE GLENN
|
TRUEX SAMANTHA
|
PETERS ROBERT T
| +1
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8
The World Federation of Hemophilia World Bleeding Disorders Registry : insights from the first 10,000 patients
enthalten in:
Research and practice in thrombosis and haemostasis
| 2023
von
Coffin, D.
|
Gouider, E.
|
Konkle, B.
| +105
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9
FACTOR VIII CHIMERIC AND HYBRID POLYPEPTIDES, AND METHODS OF USE THEREOF
enthalten in:
Europäisches Patentamt
| 2023
von
DUMONT JENNIFER A
|
LOW SUSAN
|
BITONTI ALAN J
| +9
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10
Hemophilia gene therapy : first, do no harm
enthalten in:
Journal of thrombosis and haemostasis : JTH
| 2023
von
Valentino, L.
|
Kaczmarek, R.
|
Pierce, G.
| +5
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