Results 171 to 180 of about 50,045 (313)

Immunogenicity assessment of concizumab in patients with Hemophilia A and b: Assays developed to measure anti-drug antibodies and integrated results from clinical trials [PDF]

Luis Perez Tosar, Thomas Porstmann, Lone Hummelshøj Landsy   +2 more
openalex   +1 more source

Very low dislocation rate and good clinical outcome after Bereiter trochleoplasty and additional procedures following the Copenhagen patella–femoral instability algorithm: One‐ and two‐years outcomes from a consecutive cohort of 368 cases

Knee Surgery, Sports Traumatology, Arthroscopy, Volume 34, Issue 1, Page 52-63, January 2026.
Abstract Purpose Patello–femoral instability (PFI) is often caused by predisposing factors, with trochlea dysplasia (TD) as the most prominent. Untreated patellar instability leads to impaired function and an increased risk of patellofemoral osteoarthritis.
Christian Dippmann, Volkert Siersma, Simone Rechter, Kristoffer W. Barfod, Peter Lavard   +4 more
wiley   +1 more source

Evaluation of kidney function and damage in patients with hemophilia B-insights from the B-Natural study. [PDF]

Res Pract Thromb Haemost
Astermark J, LeBeau P, Christensson A, Lethagen S, Berntorp E, Shapiro AD, B-Natural Study investigators.   +6 more
europepmc   +1 more source

Emicizumab in Previously Untreated Patients and Minimally Treated Patients With Hemophilia A: A Comparative Study Between Two International Cohorts

Pediatric Blood &Cancer, Volume 73, Issue 1, January 2026.
ABSTRACT Background Hemophilia A (HA) is a rare bleeding disorder caused by coagulation factor VIII (FVIII) deficiency. Prophylactic FVIII replacement therapy is essential for preventing bleeds, but it carries a risk of inhibitor development, especially in previously untreated and minimally treated patients (PUPs and MTPs, respectively).
Sarina Levy‐Mendelovich, Tlalit Barhod, Ivan Budnik, Jonathan Lancashire, Jesal Patel, Assaf Arie Barg, Einat Avishai, Rima Dardik, Tami Brutman Barazani, Tami Livnat, Jayashree Motwani, Gili Kenet   +11 more
wiley   +1 more source

Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B. [PDF]

N Engl J Med
Reiss UM, Davidoff AM, Tuddenham EGD, Chowdary P, McIntosh J, Muczynski V, Journou M, Simini G, Ireland L, Mohamed S, Riddell A, Pie AJ, Hall A, Quaglia A, Mangles S, Mahlangu J, Haley K, Recht M, Shen YM, Halka KG, Fortner G, Morton CL, Gu Z, Hayden RT, Neufeld EJ, Okhomina VI, Kang G, Nathwani AC.   +27 more
europepmc   +1 more source

Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system [PDF]

, 2016
Frank DeRosa, Braydon C. Guild, Shrirang Karve, L Smith, Kevin T. Love, J. Robert Dorkin, Kevin Kauffman, J Zhang, Barak Yahalom, Daniel G. Anderson, Michael W. Heartlein   +10 more
openalex   +1 more source

Treatment patterns and bleeding outcomes in persons with severe hemophilia A and B in a real-world setting [PDF]

, 2020
Cihan Ay, Leonard Perschy, Judit Rejtő, Alexandra Kaider, Ingrid Pabinger   +4 more
openalex   +1 more source

Lipid Nanoparticles for Delivery of CRISPR Gene Editing Components

Small Methods, Volume 10, Issue 2, 22 January 2026.
The review presents a comprehensive overview of each component of lipid nanoparticles（LNPs）and their effects on editing efficiency. It specifically highlights strategies for achieving non‐liver delivery, aiming for broader applications in gene editing. Furthermore, this review summarizes the applications of LNPs in gene editing and offers insights for ...
Fan Wu, Nei Li, Yudian Xiao, Rohan Palanki, Hannah Yamagata, Michael J. Mitchell, Xuexiang Han   +6 more
wiley   +1 more source

Factor IX-Padua AAV gene therapy in hemophilia B: phases 1/2 and 3 trials. [PDF]

Nat Med
Xue F, Ju M, Zhu T, Zhou Z, Sun J, Yang L, Yan Z, Zhou H, Du X, Zheng C, Zheng J, Wu X, Du Z, Jiang W, Yang C, Xiao X, Liu W, Yang R, Zhang L.   +18 more
europepmc   +1 more source

A Multicenter Clinical Study of Monoclonal Antibody-Purified Coagulation Factor IX Concentrate (GA-1013) in Treatment of Hemophilia B

, 1991
Hiromu Fukui, Yutaka Yoshida, Yasuyuki Endo, Kazuo Mori, Norio Kobayashi, Juzo Matsuda, M Fujimaki, Minoru Inagaki, Kaneo Yamada, Kojiro Honda, Akira Shibata, Hoyu Takahashi, N Sakuragawa, T Kainuma, Joichi NOMURA, Noriyuki KAWASAKI, J Mimaya, Akira Yoshioka, Shigeru Shirakawa, Katsumi Deguchi, Keiichiro Yoshioka, Hisao Ikari, Eizo Kakishita, Noboru Takata, Akira Shirahata, Kunio Kishida   +25 more
openalex   +2 more sources