Results 221 to 230 of about 113,823 (359)

Balancing Promise and Peril: Hemophilia Gene Therapy Insights. [PDF]

IUBMB Life
Akula S, Borroni E, Cottonaro A, Follenzi A, Merlin S.   +4 more
europepmc   +1 more source

Hemophilia and prophylaxis [PDF]

, 2012
Rolf Ljung
openalex   +1 more source

Real‐World Effectiveness and Safety of Damoctocog Alfa Pegol in Severe and Nonsevere Patients With Hemophilia A From the Prospective, Multinational, Ongoing HEM‐POWR Study

European Journal of Haematology, Volume 116, Issue 2, Page 148-159, February 2026.
ABSTRACT Objectives To assess the effectiveness and safety of damoctocog alfa pegol in patients with severe and nonsevere hemophilia A in the fifth interim analysis of the ongoing HEM‐POWR study. Methods HEM‐POWR (NCT03932201) is a multinational, Phase 4, prospective observational study.
Mark T. Reding, María Teresa Alvarez Román, Giancarlo Castaman, Maissaa Janbain, Tadashi Matsushita, Karina Meijer, Kathrin Schmidt, Johannes Oldenburg   +7 more
wiley   +1 more source

PB2654: RARE BLEEDING DISORDERS: LONG-TERM EXPERIENCE FROM A HEMOPHILIA CENTER

HemaSphere, 2023
Antonios Meimaridis, Sofia Chissan, Nikolaos Kotsiou, Eleni Gavriilaki, Eleni Moka, Sofia Vakalopoulou   +5 more
doaj   +1 more source

66. Effective Treatment of Hemophilia A Using Hematopoietic Stem Cell Transplantation and a Bioengineered Factor VIII Transgene [PDF]

, 2012

openalex   +1 more source

Loss of FVIII Expression by Possible Interlinked Immune and Cellular Stress Response in Hemophilia A Mice Treated with AAV Gene Therapy

, 2021
Christopher S. Rogers, Thaís B. Bertolini, Roland W. Herzog   +2 more
openalex   +2 more sources

Predicting FVIII Activity in Patients Who Use Recombinant FVIII Fc Fusion Protein for Prophylaxis and Treatment of Bleeding Episodes [PDF]

, 2014
Allen, G, Li, L, Mahlangu, JN, Pasi, J, Pierce, GF, Quon, DV, Shapiro, AD, Zhu, Y   +7 more
core  

Acquired Hemophilia

Internal Medicine, 2015
Kenji, Kimura, Akira, Kuriyama, Naoki, Kuninaga, Akira, Sasaki   +3 more
openaire   +3 more sources

PB1544 Bleeding Manifestations in Mothers with Hemophilia (Before Knowing Their Diagnosis): A Retrospective Analysis

, 2023
B. Diaz Jordan
openalex   +1 more source

Health care resources and costs associated with delivering gene therapy for hemophilia in clinical practice. [PDF]

Res Pract Thromb Haemost
Prameyllawati DM, Mussert CMA, Oude Voshaar MAH, Lingsma HF, Cnossen MH, Coppens M, Meijer K, van der Valk PR, Leebeek FWG, Ten Ham RMT.   +9 more
europepmc   +1 more source