Results 51 to 60 of about 118,583,499 (297)

Multiple criteria decision analysis for therapeutic innovations in a hemophilia care center: A pilot study of the organizational impact of innovation in hemophilia care management

, 2022
BACKGROUND: Several innovative drugs liable to lead to changes in healthcare organization are or soon will be available for the management of hemophilia.
Beny, Karen, Dubromel, Amélie, Gay, Valérie, Carrouel, Florence, du Sartz de Vigneulles, Benjamin, Negrier, Claude, Dussart, Claude   +6 more
core   +1 more source

Prime editing in neuropsychiatric disorders: From mutation‐specific target selection to clinical translation

Neuroprotection, EarlyView.
Abstract Prime editing, a novel clustered regularly interspaced short palindromic repeats (CRISPR)‐based technology, fuses a reverse transcriptase (RT) to an engineered CRISPR‐associated protein 9 (Cas9) and uses a prime editing guide RNA (pegRNA)‐encoded template.
Tianshan Ji, Yuan Zhang, Jinyi Zhao, Yiping Lu, Chengkun Wang   +4 more
wiley   +1 more source

Hemophilia A: An Ideal Disease for Prenatal Therapy

Prenatal Diagnosis, EarlyView.
ABSTRACT Hemophilia A (HA) is the most common inherited coagulation defect. Current state‐of‐the‐art treatment consists of frequent administration of prophylactic infusions of coagulation factor VIII (FVIII) protein or bispecific antibodies that replace the cofactor function of FVIIIa to maintain hemostasis. However, these treatments are far from ideal,
Christopher D. Porada, Anthony Atala, Graça Almeida‐Porada   +2 more
wiley   +1 more source

Gene Therapy for Hemophilia A: A Mixed Methods Study of Patient Preferences and Shared Decision-Making

, 2023
Jacqueline Limjoco,1 Courtney D Thornburg1,2 1Hemophilia and Thrombosis Treatment Center, Rady Children’s Hospital San Diego, San Diego, CA, USA; 2Department of Pediatrics, UC San Diego School of Medicine, La Jolla, CA, USACorrespondence: Courtney D ...
Thornburg CD, Limjoco J
core  

Child Health Challenges, Ecosystem Mismatches, and the Case for State‐Level Policy Innovation

The Milbank Quarterly, EarlyView.
Policy Points From 2011‐2023, US children's health deteriorated across >170 indicators. Chronic conditions rose from 25.8% to 31%, risk of anxiety tripled, and autism and learning disability more than doubled. Policies based on biomedical models have failed to address these trends.
NEAL HALFON, SHIRLEY RUSS
wiley   +1 more source

The association between unemployment and treatment among adults with hemophilia

Research and Practice in Thrombosis and Haemostasis
Background: People with hemophilia often experience pain and suffer from comorbidities related to their bleeding disorder. Consequently, unemployment due to disability is prevalent among people with hemophilia. Objectives: To explore associations between
Christian Qvigstad, Lars Q. Sørensen, Geir E. Tjønnfjord, Pål André Holme, Ingrid Pabinger, Cedric Hermans, Roseline d’Oiron, Robert Klamroth, Johannes Oldenburg, Natascha Marquardt, Peter Staritz, Olga Katsarou, Uri Martinowitz, Aharon Lubetsky, Gili Kenet, Annarita Tagliaferri, Maria Elisa Mancuso, Roger Schutgens, Pål André HolmE, Jerzy Windyga, Irena Zupan, Victor Jimenez Yuste, Ramiro Nunez, Philippe de Moerloose, Erik Berntorp, Jan Astermark, Campbell Tait, Gerry Dolan   +27 more
doaj   +1 more source

A Rare Case of Acquired Haemophilia A Following Sigmoid Adenocarcinoma: Case Report and Literature Review

Asia-Pacific Journal of Clinical Oncology, EarlyView.
Acquired haemophilia A (AHA) is a rare complication of malignancy. We report a case of AHA secondary to colorectal cancer which presented as post‐operative bleeding, progressive activated partial thromboplastin time prolongation, precipitous factor VIII (FVIII) decline and inhibitor development.
Benjamin Reardon, Harini Haran, Jennifer Curnow   +2 more
wiley   +1 more source

Plain language summary: How safe and effective is long-term treatment with intravenous plasminogen concentrate for people with type 1 plasminogen deficiency?

Therapeutic Advances in Hematology
Summary What is this study about? Type 1 plasminogen deficiency (PLGD-1 for short) is a long-term, ultra-rare condition where the body does not make enough of a protein called plasminogen.
Amy D. Shapiro, Heather McDaniel, Robert W. Decker, Charles Nakar, Jeremy Lorber, Neelam Thukral, Joseph M. Parker, Karen Thibaudeau   +7 more
doaj   +1 more source

Factor 8 Gene Mutation Spectrum of 270 Patients with Hemophilia A: Identification of 36 Novel Mutations

Turkish Journal of Hematology, 2020
Objective: Hemophilia A (HA) is the most severe X-linked inherited bleeding disorder caused by hemizygous mutations in the factor 8 (F8) gene. The aim of this study is to determine the mutation spectrum of the F8 gene in a large HA cohort from Turkey ...
Tahir Atik, Esra Işık, Hüseyin Onay, Bilçağ Akgün, Moharram Shamsali, Kaan Kavaklo, Melike Evim, Gülen Tüysüz, Namık Yaşar Özbek, Fahri Şahin, Zafer Salcıoğlu, Canan Albayrak, Yeşim Oymak, Ekrem Ünal, Fatma Burcu Belen, Ebru Yılmaz Keskin, Can Balkan, Birol Baytan, Alphan Küpesiz, Vildan Culha, Tuba Nur Tahtakesen, Adalet Meral Güneş, Ferda Özkınay   +22 more
doaj   +1 more source

Hemophilia, HIV and AIDS : perspectives in living : workshop study guide / John (Jack) R. McDonald, Jonathan Cooper

, 1991
Prepared by the World Federation of Hemophilia in collaboration with the World Health Organization79 ...
World Health Organization, Copper, Jonathan, McDonald, John R, World Federation of Hemophilia   +3 more
core