Results 111 to 120 of about 13,000,271 (286)

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

Gene Therapy, 2016
DNA-based gene therapy has considerable therapeutic potential, but the challenges associated with delivery continue to limit progress. Messenger RNA (mRNA) has the potential to provide for transient production of therapeutic proteins, without the need ...
F. Derosa, Braydon Charles Guild, S. Karve, Lynelle P. Smith, Jiasheng Zhang, B. Yahalom, M. Heartlein, Kerry R. Love, J. R. Dorkin, Kevin J. Kauffman, Daniel G. Anderson   +10 more
semanticscholar   +1 more source

Real‐World Unmet Needs of Patients With Haemophilia A and Haemophilia B With or Without Inhibitors: End‐of‐Study Results From the explorer6 Non‐Interventional Study

Haemophilia, EarlyView.
ABSTRACT Introduction Haemophilia is associated with high disease and treatment burdens. Prospective evaluation of data from patients with haemophilia helps understand and define unmet needs, optimise treatment and improve healthcare outcomes. Aim To present end‐of‐study data from explorer6 (NCT03741881), a prospective, non‐interventional study across ...
Allison P. Wheeler, Aby Abraham, Chris Barnes, Renée Brown Frandsen, Roseline d'Oiron, Hermann Eichler, Kingsley Hampton, Francisco José López‐Jaime, Chuhl Joo Lyu, Camila Martins Mazini Tavares, Keiji Nogami, Christopher Sutton, Jerzy Windyga, Bulent Zulfikar, Giancarlo Castaman   +14 more
wiley   +1 more source

Prophylaxis in Hemophilia

Clinical and Biomedical Research, 2023
Hemophilia is an inherited X-linked coagulopathy defined by a deficiency or abnormality in the clotting function of factor VIII (Hemophilia A) or factor IX (Hemophilia B).
Júlia Plentz Portich, Dayenne Catelli, Ebellins Tabares Calvache, Cristiane Seganfredo Weber, Daniel Sander Hoffmann, Guilherme Rasia Bosi, Leo Sekine, Lucia Mariano da Rocha Silla   +7 more
doaj  

A Girl with Hemorrhagic Diathesis in a Family of Mild Hemophilia B (Female Hemophilia B?)

Blood & Vessel, 1979
Female carriers of hemophilia are usually asymptomatic. Female hemophilia B or symptomatic carriers of hemophilia B are extremely rare and so far only 16 cases have been reported.We report a girl with hemorrhagic diathesis in a family of mild hemophilia B, who was suspected female hemophilia B.
Takeshi Nagao, Atsuo Iizuka
openaire   +3 more sources

European Principles of Nursing Care for Persons With Inherited Bleeding Disorders

Haemophilia, EarlyView.
ABSTRACT Introduction A nurse is one of the core members of the multidisciplinary haemophilia team. However, what people with inherited bleeding disorders (PwBD) can expect from them has not yet been formally defined. Aim This project, initiated by the EAHAD Nurses Committee, aimed to establish Principles of Nursing Care (PoNC) for PwBD to promote high‐
Nanda Uitslager, Mary Kavanagh, Maj Birkedal, Linda Myrin‐Westesson, the EAHAD Nurses Committee   +4 more
wiley   +1 more source

Hemophilia B in a male with a four-base insertion that arose in the germline of his mother [PDF]

, 1989
C. D. K. Bottema, Rhett P. Ketterling, H.I. Cho, Steve S. Sommer   +3 more
openalex   +1 more source

Enhancing Engagement to Support Shared Decision Making Within Haemophilia: Development of Materials for People With Haemophilia, Caregivers and Healthcare Professionals

Haemophilia, EarlyView.
ABSTRACT Introduction Treatment decision making can be complex and challenging for people with haemophilia (PwH) and their caregivers. A shared decision‐making (SDM) approach can ensure that decisions about treatment and care incorporate patients’ beliefs, values, and preferences.
Laura Meade, Haowei (Linda) Sun, Robert J. Klaassen, Roxana Bahar, Shade Olatunde, Michelle Santos, Sheri vanGunst   +6 more
wiley   +1 more source

A Dutch family with moderately severe hemophilia B (Factor IX_Heerde) has a missense mutation identical to that of factor lX_{London 2} [PDF]

, 1989
S R Poort, E Briët, R M Bertina, P H Reitsma   +3 more
openalex   +1 more source

Systemic lupus erythematosus caused acquired hemophilia B without evidence of factor IX inhibitors

Rheumatology &Autoimmunity, EarlyView.
Lifang Wang, Yan Ding, Lei Wang, Lianjie Shi   +3 more
wiley   +1 more source

Developing a Two‐Sided Decision Box to Facilitate Shared Decision‐Making for Switching From Conventional to Pharmacokinetic‐Tailored Prophylaxis in Haemophilia

Haemophilia, EarlyView.
ABSTRACT Introduction Decision‐making in haemophilia is challenging due to the small evidence base, disease heterogeneity, and inter‐patient variability. Shared decision‐making (SDM) supports patient‐clinician decisions. Aim Creation of a two‐sided decision box facilitating SDM for haemophilia patients switching from conventional (weight‐based) to ...
Arun Keepanasseril, Athena Mancini, Megan S. Lowe, Noella Noronha, Alfonso Iorio   +4 more
wiley   +1 more source