Results 41 to 50 of about 50,045 (313)

Serum Vitamin D in Children with Hemophilia A and Its Association with Joint Health and Quality of Life

Hematology Reports
Background/Objectives: Hemophilia A is an X-linked recessive illness produced by a deficiency of coagulation factor VIII. This study aimed to evaluate serum vitamin D in hemophilic pediatric patients and its correlation with joint health and quality of ...
Aida M. S. Salem, Takwa Mohamed AbdEltwwab, Hanan Hosni Moawad, Marwa O. Elgendy, Reham S. Al-Fakharany, Ahmed Khames, Mohamed Hussein Meabed   +6 more
doaj   +1 more source

Clinical, humanistic, and economic burden of severe hemophilia B in the United States: Results from the CHESS US and CHESS US+ population surveys

Orphanet Journal of Rare Diseases, 2021
Background Hemophilia B is a rare congenital bleeding disorder that has a significant negative impact on patients’ functionality and health-related quality of life.
Tom Burke, Sohaib Asghar, Jamie O’Hara, Eileen K. Sawyer, Nanxin Li   +4 more
doaj   +1 more source

The frequency of complications in a cohort of patients diagnosed with hemophilia A and hemophilia B receiving prophylactic treatment in Colombia: A retrospective noninterventional study.

PLoS ONE, 2023
IntroductionHemophilia A and B are disorders associated with the deficit of coagulation factors VIII and IX.ObjectiveWas to determine the incidence of complications in a cohort of patients diagnosed with moderate and severe hemophilia A or B under ...
Jorge E Machado Alba, Juan David Wilches-Gutierrez, Diana Rocio Arias-Osorio, Juan Manuel Reyes, Maria Lourdes Nakandakari, Harrison David Ospina-Arzuaga, Andres Gaviria-Mendoza, Natalia Castaño-Gamboa, Luis Fernando Valladales-Restrepo, Manuel E Machado-Duque   +9 more
doaj   +1 more source

Progress in the use of adeno-associated viral vectors for gene therapy [PDF]

, 2004
The development of safe and efficient gene transfer vectors is crucial for the success of gene therapy trials. A viral vector system promising to meet these requirements is based on the apathogenic adeno-associated virus (AAV-2), a member of the ...
Braun-Falco, M., Buning, H., Hallek, M.
core   +1 more source

Potent Liver‐Tropic mRNA Lipid Nanoparticles: ApoE‐Mediated Delivery Through a Low‐Density Lipoprotein Receptor Independent Uptake Mechanism

Advanced Materials, EarlyView.
Helper and ionizable lipids play a crucial role in determining ApoE binding and subsequent liver tropism and LDLR‐mediated uptake. Ionizable lipids primarily govern the LDLR‐independent uptake pathway. This complementary interplay between lipid components ultimately governs LNP delivery performance and therapeutic efficacy in the liver.
Ashish Sarode, Christian Ortiz, Tadeh Derstepanian, Natalia Vargas‐Montoya, Priyal Patel, Nikita Khadse, Saikat Manna, Ryan Landis, Joseph Skaleski, Lianne Boeglin, Hongfeng Deng, Anusha Dias, Hong Wang, Debora Barreiros Petropolis, Barak Yahalom, Shrirang Karve, Frank DeRosa   +16 more
wiley   +1 more source

A Case of Acquired Hemophilia A and Congenital Hemophilia B

Cureus, 2022
Congenital hemophilia B is a rare, inherited X-linked bleeding disorder caused by a deficiency of factor IX (FIX). Acquired hemophilia A is a rare, acquired bleeding disorder which presents as new onset bleeding in older adults due to the development of autoantibodies against factor VIII (FVIII).
Fortier, Julia C, Pang, Shiyi S, Amofa-Ho, Sam, Harris, Neil S, Zumberg, Marc   +4 more
openaire   +2 more sources

PRODUCTION OF HIGH LEVELS OF TRANSGENC FACTOR IX WITHOUT GENE RESCUE, AND ITS THERAPEUTIC USES [PDF]

, 2011
A non-human transgenic mammalian animal, as described above, contains an exogenous double stranded DNA sequence stably integrated into the genome of the animal, which comprises cis-acting regulatory units operably linked to a DNA sequence encoding human ...
Cooper, Julian D., O\u27Sickey, Tanya K., Velander, William Hugold   +2 more
core   +1 more source

An assessment of the relative influence of pain coping, negative thoughts about pain, and pain acceptance on health-related quality of life among people with hemophilia [PDF]

, 2009
Many people with hemophilia are affected by chronic arthritic joint pain as well as acute bleeding pain. In this cross-sectional study, 209 men with hemophilia A or B completed the Hemophilia Pain Coping Questionnaire (HPCQ), the Chronic Pain Acceptance ...
Barakat, Baron, Barry, Choiniere, Elander, Elander, Esteve, Fischer, Fischer, Georgina Robinson, Gil, Gil, Hays, Hays, James Elander, Jensen, Jensen, John Morris, Kathryn Mitchell, Kratz, McCracken, McCracken, McCracken, McCracken, McCracken, McCracken, McCracken, McHorney, McHorney, Miners, Miners, Preacher, Roberts, Romano, Rosenstiel, Royal, Ruta, Santavirta, Solovieva, Solovieva, Stansfield, Szende, Trippoli, Turner, Turner, Vowles, Vowles, Vowles   +47 more
core   +1 more source

Traceless Regulation of Genetic Circuitry

Advanced Science, EarlyView.
Energy‐based, as opposed to molecular, control offers unprecedented improvements in key circuit parameters. This review summarizes the fundamentals of such traceless switches, categorizes them by trigger modalities, and compares and contrasts distinct advantages as well as shortcomings of each kind.
Gokberk Unal, Martin Fussenegger
wiley   +1 more source

Haemophilia and joint disease: pathophysiology, evaluation and management [PDF]

, 2011
In patients with haemophilia, regular replacement therapy with clotting factor concentrates (prophylaxis) is effective in preventing recurrent bleeding episodes into joints and muscles.
Berntorp, Erik, Knobe, Karin
core   +3 more sources