Results 91 to 100 of about 118,583,499 (297)

RNAi for the Treatment of People with Hemophilia: Current Evidence and Patient Selection

, 2023
Sara Boyce,1 Savita Rangarajan2 1Haemophilia Comprehensive Care Centre, University Hospital Southampton, Southampton, UK; 2Faculty of Medicine, University of Southampton, Southampton, UKCorrespondence: Sara Boyce, Email sara.boyce@uhs.nhs.ukAbstract ...
Rangarajan S, Boyce S
core  

Italian Patients Journey for Gene Therapy in Haemophilia A

Haemophilia, EarlyView.
ABSTRACT Introduction Gene therapy (GT) provides sustained FVIII levels without repeated infusions in Hemophilia A (HA) patients thus overcoming a major limitation of replacement therapy. However, issues remain e.g., patient selection criteria, duration and variability of transgene expression, quality of life and long‐term safety.
Giovanni Di Minno, Gaia Spadarella, Alessandra Borchiellini, Anna Borrelli, Andrea Buzzi, Giancarlo Castaman, Michele Cecchi, Raimondo De Cristofaro, Vincenzo La Mura, Angelo Lupi, Giuseppe Malcangi, Maria Elisa Mancuso, Maria Francesca Mansueto, Cristina Santoro, Rita Carlotta Santoro, Luigi Piero Solimeno, Annarita Tagliaferri, Ezio Zanon, Isabella Garagiola, Flora Peyvandi   +19 more
wiley   +1 more source

Nonneutralizing antibodies in Nordic persons with moderate hemophilia A and B (the MoHem study)

Research and Practice in Thrombosis and Haemostasis
Background: The impact of nonneutralizing antibodies (NNAs) in moderate hemophilia is elusive. Objectives: To explore the presence of NNAs in Nordic persons with moderate hemophilia A (MHA) and B (MHB) in relation to treatment modality, clinical outcome,
Ragnhild J. Måseide, MD, PhD, Erik Berntorp, MD, PhD, Jan Astermark, MD, PhD, Anna Olsson, MD, PhD, Maria Bruzelius, MD, PhD, Tony Frisk, MD, PhD, Vuokko Nummi, MD, PhD, Riitta Lassila, MD, PhD, Karin Strandberg, MD, PhD, Geir E. Tjønnfjord, MD, PhD, Pål A. Holme, MD, PhD   +10 more
doaj   +1 more source

Treatment adherence in hemophilia

, 2017
Courtney D Thornburg,1 Natalie A Duncan2 1Hemophilia and Thrombosis Treatment Center, Rady Children’s Hospital, San Diego, CA, 2Indiana Hemophilia and Thrombosis Center, Indianapolis, IN, USA Abstract: Prophylactic clotting-factor regimens reduce
Thornburg CD, Duncan NA
core  

Challenges in prophylactic therapy with Emicizumab in patients with hemophilia A: Focus on monitoring tests

Romanian Journal of Laboratory Medicine
This study presents a transversal investigation that we performed at Fundeni hospital (Bucharest, Romania) into the therapeutic benefits and efficacy of Emicizumab, a non-factor therapy, in the context of hemophilia A.
Brinza Melen, Uscatescu Valentina, Hemcinschi Mihai, Chiriac Elisabeta, Gherghe Georgiana, Coriu Daniel   +5 more
doaj   +1 more source

Recent Advances in the Treatment of Hemophilia: A Review

, 2021
Emanuela Marchesini,1 Massimo Morfini,2 Leonard Valentino3,4 1Hemophilia Centre, SC Vascular and Emergency Department, University of Perugia, Perugia, Italy; 2Italian Association of Haemophilia Centres (AICE), Naples, Italy; 3National Hemophilia ...
Marchesini E, Valentino L, Morfini M
core  

Lack of Inhibitor Development in Previously Treated People With Haemophilia Switching Factor Products—Final Results of Athn 2: Factor Switching Study

Haemophilia, EarlyView.
Erin Cockrell, Martin Chandler, Tammuella Chrisentery‐Singleton, Stacy E. Croteau, Nabil Daoud, Michael F. Guerrera, M. Nikki Hirsh, Janna Journeycake, Roshni Kulkarni, Susan U. Lattimore, Catherine McGuinn, Margaret Ragni, Michael Recht, Ulrike M. Reiss, Robert Sidonio Jr., Cliffford M. Takemoto, Michael Tarantino, Annette von Drygalski, Ellis J. Neufeld   +18 more
wiley   +1 more source

Use of an Oral Health‐Related Quality of Life Instrument to Measure Unmet Dental Care Needs in Adults With Inherited Bleeding Disorders

Haemophilia, EarlyView.
ABSTRACT Introduction Preventive dental care is vital for individuals with bleeding disorders to reduce the need for potentially invasive procedures. Although dental care is a mandated function of U.S. federally supported hemophilia treatment centers (HTCs), access to dental care is widely variable.
Miguel A. Escobar, Megan M. Ullman, Joanna Larson, Michael M. Chan, Marisela Trujillo   +4 more
wiley   +1 more source

Real‐world use of emicizumab in Chinese children with hemophilia A: Retrospective data from a comprehensive care center

Pediatric Investigation
Importance Emicizumab (EMI) is efficacious and safe for hemophilia A (HA) prophylaxis. However, its high cost poses a challenge in China. Objective To explore the possibility of using reduced‐dosage EMI in Chinese HA children.
Qianqian Mao, Zhenping Chen, Guoqing Liu, Gang Li, Yingzi Zhen, Xiaoling Cheng, Zekun Li, Wanru Yao, Di Ai, Zhengping Li, Nan Wang, Man‐Chiu Poon, Runhui Wu   +12 more
doaj   +1 more source

Physical Activity and Sedentary Behaviour in Children With Haemophilia

Haemophilia, EarlyView.
ABSTRACT Introduction Physical activity (PA) is vital in child development, while inactivity remains a leading contributor to global mortality. Therapeutic advances have enabled greater PA in boys with haemophilia (BwH), yet robust UK data on objectively measured PA and sedentary behaviour (SB) across severities are limited.
Melanie Bladen, Lucy Alderson, Eleanor Main, Mario Cortina‐Borja   +3 more
wiley   +1 more source