Results 161 to 170 of about 13,086,034 (342)

Effectiveness of Octocog Alfa (BAY 81‐8973) to Treat People With Haemophilia A Enrolled in the ATHNdataset, Including Under‐Represented Subgroups

European Journal of Haematology, Volume 115, Issue 5, Page 484-492, November 2025.
ABSTRACT Objectives To evaluate the real‐world effectiveness of octocog alfa (BAY 81‐8973; Kovaltry) in people with haemophilia A (PwHA), including under‐represented subgroups. Methods We retrospectively analysed the American Thrombosis and Hemostasis Network (ATHN) dataset to identify the characteristics, treatment/clinical histories and annualised ...
Martin Chandler, Thomas Moulton, Lena Charafi, Jessica Charlet, Michael Recht   +4 more
wiley   +1 more source

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.

New England Journal of Medicine, 2011
A. Nathwani, E. Tuddenham, S. Rangarajan, Cecilia Rosales, J. McIntosh, D. Linch, P. Chowdary, A. Riddell, Arnulfo Pie, C. Harrington, J. O’Beirne, Keith A. Smith, J. Pasi, B. Glader, P. Rustagi, C. Ng, M. Kay, Junfang Zhou, Yunyu Spence, C. Morton, J. Allay, J. Coleman, S. Sleep, J. Cunningham, D. Srivastava, E. Basner-Tschakarjan, F. Mingozzi, K. High, J. Gray, U. Reiss, A. Nienhuis, A. Davidoff   +31 more
semanticscholar   +1 more source

Standardization of Terminology, Definitions, and Outcome Criteria for Bleeding in Hereditary Hemorrhagic Telangiectasia: International Consensus Report

American Journal of Hematology, Volume 100, Issue 10, Page 1813-1827, October 2025.
ABSTRACT Hereditary hemorrhagic telangiectasia (HHT, Osler‐Weber‐Rendu disease) is the second most common inherited bleeding disorder worldwide, affecting approximately 1 in 5000 people. Development of disease‐modifying and efficacious hemostatic agents to treat HHT has finally begun after decades without such medical therapies.
Hanny Al‐Samkari, Raj S. Kasthuri, Hans‐Jurgen Mager, Jenny Y. Zhou, Marcelo M. Serra, Bethany T. Samuelson‐Bannow, Layla N. Van Doren, Jay F. Piccirillo, Marianne S. Clancy, Keith R. McCrae, Sonia M. Thomas, Antoni Riera‐Mestre, Allyson M. Pishko, Sarah Sewaralthahab, James R. Gossage, Vivek N. Iyer, Cedric Hermans, Adrienne Hammill, Ingrid Winship, Meir Mei‐Zahav, Annette von Drygalski, Scott Olitsky, Marie E. Faughnan   +22 more
wiley   +1 more source

Splice junction mutations in factor IX gene resulting in severe hemophilia B [PDF]

, 1991
S.H. Chen, Miqin Zhang, Arthur R. Thompson, Gordon L. Bray, C. Ronald Scott   +4 more
openalex   +1 more source

cGMP Recombinant FIX for IV and Oral Hemophilia B Therapy [PDF]

, 2005
Three specific aims are proposed: Specific Aim # 1. Process engineer and scale-up the recovery and purification of transgenic recombinant human Factor IX.
Dernell, William, KEY PERSON, Julian, Cooper, LEAD INVESTIGATOR, Manning, Mark, LEAD INVESTIGATOR, Meagher, Michael, LEAD INVESTIGATOR, Mohanan, Paul, LEAD INVESTIGATOR, Nichols, Timothy, LEAD INVESTIGATOR, Stephan, Abramson, LEAD INVESTIGATOR, Swaanson, Todd, KEY PERSON, Van Cott, Kevin E., Velander, William H., PRINCIPAL INVESTIGATOR   +9 more
core   +1 more source

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

New England Journal of Medicine, 2014
A. Nathwani, U. Reiss, E. Tuddenham, Cecilia Rosales, P. Chowdary, J. McIntosh, M. della Peruta, Elsa Lhériteau, Nishali Patel, D. Raj, A. Riddell, J. Pie, S. Rangarajan, D. Bevan, M. Recht, Yu-min P Shen, K. Halka, E. Basner-Tschakarjan, F. Mingozzi, K. High, J. Allay, M. Kay, C. Ng, Junfang Zhou, M. Cancio, C. Morton, J. Gray, D. Srivastava, A. Nienhuis, A. Davidoff   +29 more
semanticscholar   +1 more source

Technical Note and Short Review: A Challenging Case of a Premature Newborn Presenting With ICH, IVH, Ventriculitis, and Hydrocephalus

Clinical Case Reports, Volume 13, Issue 10, October 2025.
ABSTRACT Premature infants are at a heightened risk of intracerebral or intraventricular hemorrhages (IVH), which often lead to posthaemorrhagic hydrocephalus and necessitate the placement of a ventriculoperitoneal (VP) shunt. Here, we present a case involving suspected ICH, which led to multiloculated hydrocephalus, recurrent ventriculitis, and ...
Hannes Egermann, Christian Knorr, Basheer Al‐Shameri, Adolf Müller, Markus Denzinger, Stephan Lackermair   +5 more
wiley   +1 more source

Clinical audit of inherited bleeding disorders in a developing country

Indian Journal of Pathology and Microbiology, 2010
Objective: We did a clinical audit to determine the status of coagulation disorders in a hemophilia care center in Pakistan. Setting: Fatimid foundation blood bank and hematological diseases center, Lahore.
Sajid Raihan, Khalid Safoorah, Mazari Nazish, Azhar Waleed, Khurshid Mohammad   +4 more
doaj  

Quality of Life and Treatment Satisfaction in People With Haemophilia on Fitusiran Prophylaxis: Evidence From a Subset of ATLAS‐OLE Trial Participants Mainly From India

Haemophilia, EarlyView.
Alok Srivastava, Savita Rangarajan, Cecil Ross, Shariq Ali, Shauna Andersson, Laurel A. Menapace, Marja Puurunen, Marion Afonso   +7 more
wiley   +1 more source

INDUCTION OF TOLERANCE BY ORAL ADMINISTRATION OF FACTOR VIII AND TREATMENT OF HEMOPHILA [PDF]

, 2011
Disclosed herein is a simple method for the treatment of antigen-deficiency diseases, by orally administering to a Subject a therapeutically effective amount of the deficient anti gen, wherein the antigen is not present in a liposome.
Alpan, Oral, Kamala, Tirumalai, Matzinger, Polly, Velander, William Hugold   +3 more
core   +1 more source